Category Archives: Gene Therapy

Gene Therapy: Present and Future

If our group has learned anything from these past few months of learning about field of gene therapy is that right now, no one truly has the answers to the ethical, societal and economic implications that this scientific cure could bring.

Taking a step back, we began our research at the start of it all. What is gene therapy? Through thorough research into this, we found that gene therapy is equal parts technology and medicine. Technologically, there are therapies ready to be put on the market to cure diseases such as blindness, Alzheimer’s, Parkinson’s and more but there is more to it than just technology, remember this is medicine. In the tech world, speed is everything. It is all about winning the race against smartphones, smart TV’s, AI, anything. In medicine, there is a time press that comes from the DOH in emergencies but most of the time, speedy remedies to disease put up red flags for those being treated later on. What gene therapy has done is take medicine from a personal relationship, to a big business, one that is on track to be bigger than the pharmaceutical sector. This big business is seen in one of our insights into the cost of gene therapy. The overall controversy of how one can afford to buy a gene therapy is due to the high costs that of the gene therapies that are on the market today such as the first FDA approved gene therapy Kymirah among others.

Such being the case, Dr. Mark McClellan, former FDA commissioner and current leader of the Duke-Margolis Center for Health Policy, concluded that the healthcare system is far behind the medical industry and the advances it has made so, being that patients, doctors and researchers, and insurance companies have mutually exclusive interests, it is impossible for everyone to win. The entire gene therapy field is now looking to monetize on their ability to cure what used to be deemed incurable diseases and this is where the ethics of it all come into play.

Journalist and historian, David Perry wrote, “the pro-information approach demands that everyone involved in genetic counseling have access to the best data and presents it in a value-neutral way.” In giving everyone equal access to gene therapy, this must include attainable price points, no discrimination and equal opportunity for everyone to take advantage of. The FDA is just no longer enough to control the medical and genetic communities. The ethical future of gene therapy is now beyond the typical, “this is wrong” and “this is right” because we have never had to deal with the controversial issues that we are dealing with now as a result of gene therapy. From “designer babies” to biological weapons, these are both things that health professionals and even legislators have yet to encounter scenarios such as these but because of this, getting out ahead of the possible negative implications of these two examples, is more important now than ever.

We are in a place where CRISPR can help farmers combat climate change by genetically altering their crops, incentivize football stadiums to start “DNA Days” and, lead to the insurance and pharmaceutical industries to be left in the hands of tech giants like Amazon, Apple and Google. As such, there needs to be some sort of precedent set so that none of these three scenarios gets any worse than they already have. With genetic testing companies like 23andMe and Ancestry becoming more familiar in the public eye, they are being sold to customers who have no idea that they are basically giving our their privacy rights to their own genetic information for results that are little to completely, inaccurate.

This is why education within this industry is more important now than ever. Without proper understanding, the Frightful Five, the FDA, the federal government could all have influence over every single person residing the in the United States because of those who feel it is important to send a swab of their DNA to a lab in order to see just what percentage English they really are. What people sending in their cheek swabs needs to know is that scientists are still working, to this day, trying to uncover the complex information of the millions of genes that reside in the human body. We simply do not know enough to give sufficient information to people who are just looking to see if they are carrying a genetic mutation. These 23andMe, Ancestry, and Helix tests cannot significantly provide lifesaving information and as a result, should be used with a grain of salt, or even less than that. It seems unlikely for anyone to give up the password to their email but when shipping their DNA to an unknown lab somewhere in the United States, people do not seem to show the same concern for their privacy. Thinking about the millions of people having their data breached across the country, by more companies than one, how can we be sure that this genetic information will not be next? Now even thinking ahead, the potentially 6 million more students studying harder, retaining more information, and ultimately becoming better learners because of gene therapy treatments will be ahead of the present generations. Taking it a step further, if this trend is multiplied across generations, eliminating prohibitive, cognitive disorders in students, it could reshape the entire education system with many subsequent disruptions.

With a Frightful Five company such as Amazon at the brink of breaking into the healthcare industry, it could mean positive things for gene therapy whether it investment in research, funding of drugs, therapies and clinical trials, these tech companies, although you do not want to trust with your health data, can contribute a lot to the gene therapy community. But, there needs to be a point at which the line is crossed and sure, donating genetic information for research is ethical and legal but when it is collected through your Google search history or updates on your Apple health app, there needs to be a call for change. In the end, with the Frightful Five having taken initiative within the healthcare industry to fund gene therapy programs and rebuild the healthcare system of the nation, they can push bounds in the battle for updated health services unlike anyone else can and this gap between the Frightful Five and everyone else is what needs to be addressed.

And to end on a global note, the future of gene therapy is the United States barely has a clear path ahead of it so thinking about how the rest of the world compares is a whole other thing on its own. Isolated countries such as North Korea, Russia and China make it difficult to know how exactly the countries have progressed in their biotechnological warfare, including gene therapy, and what the affects can be on the rest of the world when a country, although small compared to the US, can utilize science to become a super-powered population. It is to avoid scenarios such as the one posed by North Korea that the FDA was put in place. In order to ensure that the medicinal and agricultural sectors of the nation were being regulated and monitored, there needs to be an organization that oversees research that secure public health and safety. But, just having one country, out of hundreds with the capability to genetically alter genes and embryos with this agency in place, is not enough to guarantee that gene therapy will only be used for good.

There are a lot of things to ponder over here, but the best place to start is by educating yourself on the future that could be completely disrupted by this biotechnological innovation and then looking for ways to protect your genetic and health information, so if that means resisting buying genetic testing kits, not updating your Apple health data, or any of the other numerous ways to protect yourself, that at least is a good place to start.

Gene Therapy on the External Part of the Body

This semester, we have discussed the idea of injecting programmed/man made vectors into the body in order to help combat diseases and ailments. However, almost everything that has been discussed focuses on the inside of the body, such as diabetes, heart failure, and many other diseases. Despite the multitude of organs inside the body, most people do not associate skin as being an organ, let alone  being the largest organ in the body. This is one of the many reasons that skin is often less prioritized as an organ, and aside from putting sunblock on during the summer, some people don’t even think about their skin’s health or needs. For those with skin conditions such as junctional epidermolysis bullosa, gene therapy might be the answer to the question: do vectors and gene therapy work just as effectively on an external area? How will the body potentially reject it?

Unfortunately, the short answer is we just do not know yet. And we probably will not know for while. For treatment on the boy with junctional epidermolysis bullosa, his skin was extremely fragile and was expected to lead to more health problems throughout his life. The entire procedure was extremely expensive and tailored to his condition and genetic history. Yet, since it worked on him, the entire idea is currently batting 1.000 despite it only being tested on one person. The only definite going forward from this is that performing gene therapy on skin would have a distinct advantage since it is an entirely visible organ- a unique attribute that sets skin a part from anything other organ.

While some gene therapy patients pass away from the condition that they were originally trying to fight, the unfortunate truth is that some also pass away from the body rejecting the vectors in the system- and that makes perfect sense. Is every person’s body going to accept a foreign, man made substance that is manipulating the body from the inside? Of course not. Patients are monitored after undergoing gene therapy in order to see how the body is reacting from the gene therapy. During this process, blood, vital, and other tests are monitored to view how the body is behaving. Having the ability to visually monitor the organ is another asset in terms of detecting signs of the body rejecting the vector. Unfortunately, only time will reveal the effectiveness of this particular field of gene therapy. Here’s a video that explains how gene therapy is used for correcting skin sells:

The Ethics of Gene Editing

With the ability of scientists to genetically modify and edit one’s DNA, they have not only unlocked the possibility of curing diseases they are even diagnosed but have also opened a gateway to the unknown that offers little to no protection, security or nonthreatening state of mind. As said by Andrew Joseph, “People have access to more information about their own genes — or, in this case, about the genes of their potential offspring — than ever before. But having that information doesn’t necessarily mean it can be used to inform real-life decisions.” Gene therapy is still in its beginning stages and the reliance that people have on it being able to help them make decisions about their well being and the well being of their offspring should be taken with a grain of salt. These genetics tests or treatments are not always completely accurate and due to this, should not be significant factors when thinking about the ethical implications of a mother and father deciding what to do with their two week old fetus that is tested, and comes back positive, for a gene that could harm them in the future.

Bioethicist Alta Charo on the other hand does not see that the implications of gene therapy will affect the ethics of parents choosing “designer babies” or of the fears by governments that it will induce a new population of people against one another. As such, her comment that “genetics doesn’t tell us everything we need to know…they have tremendous influence … but we don’t have to assume that by having genetic information we will abuse the choices it facilitates,” implies that the worst case scenarios that may occur with gene therapy, may not even happen. China’s leading CRISPR researcher Dr. Lai Liangxue sees gene therapy in a similar light as the atomic bomb, noting, “I say that depends on who use it, right? Like, like, atomic bomb. That’s kind of — if you use it to make electricity, it’s good. If you use it in a bomb, it’s bad.” So, the fear that an entire new race of species of human can be created within a few decades can be seen as unfounded. There are just too many concerning factors but as can be seen from scientists whose research is the embodiment of their entire careers, they are too inclined to see the negative implications that their work can bring and instead seek to call these fears as irrational and unnecessary. These scientists are exhuming their confidence in the human race by noting how most are both rational and good but the underlying theme is, especially using Dr. Liangxue’s example of the atomic bomb, is that the human race is not all born innately good and since if there is a possibility that gene therapy can pave the way for dangerous experiments, someone will and can use it to their advantage.

In noting the comments made by Dr. Liangxue and Dr. Charo, the question then is, are these fears actually irrational and unnecessary? Because if they are, why does the DOD and Pentagon want influence over genetic treatments? Not for nothing, when a nation’s military and security sector want to track and monitor genetic treatments, the unfounded fears that scientists debate against are certainly something significant enough to deem relevant, to say the least. When the Pentagon sees genetic treatments as ways to cause injury to thousands by “agents of war,” there needs to be a wake up call within the healthcare community. The FDA is just no longer enough to prevent genetic therapy from reaching those who do evil and even with its regulation, there is no saying just how much control they will have over medical and genetic treatments. Although it can be argued that in the coming age of CRISPR and other gene therapies, the need for everyone to have the same and equal opportunity to get information will be significant in preventing the ethical and moral issues that can arise when it is not given out.  Best stated by journalist and historian, David Perry, “the pro-information approach demands that everyone involved in genetic counseling have access to the best data and presents it in a value-neutral way.”

The issues that can arise from the lack of motivation on behalf of the gene therapy community to speak on the negative effects of gene therapy because of their involvement in their own research and development of their treatments will be greater than just sidestepping the issue until more government agencies get involved.

Who’s Going to Pay for Gene Therapy?

While gene therapy has the potential to save countless people from genetic disorders, only a select few will be able to take advantage of the technology without a price reduction. The cheapest gene therapy treatment on the market today is Yescarta, which ranges from two to four hundred thousand dollars. On the high end, Glybera comes in a whopping $1 million for a single treatment. In terms of eligible patients, Yescarta has approximately 7,500 whereas Glybera has fewer than 10. In an article published by MIT Technology Review, they examined the relationship between the cost of the treatment and the number of eligible patients. In summary, the fewer number of potential patients, the higher the cost of the treatment. This math makes sense because if the R&D is roughly equal for two drugs, but one drug can only be sold to half as many people, the pharmaceutical company needs to get more revenue from each patient. The unfortunate truth is that economics makes no concessions for patients in need of treatment.

Gene therapy treatments are expensive for several reasons, one of which is the age of the technology. In any market, the first products are always the most expensive. When the newest iPhone comes out, they never lower the price. In the pharmaceutical industry especially, the first products to market are often the most expensive to produce. While a new iPhone might have a new screen or a better camera, new medicine is the product of countless hours of research and development, which can be quite costly. FDA regulations also add to the time and cost associated with developing a new medicine or treatment, this is for good reason, but expensive nonetheless.

Sure, the first to market can command high prices, but one of the largest factors affecting the price of new medicines and treatments is the deregulation of the pharmaceutical industry. Pharmaceutical companies are given a lot of liberty in terms of pricing new drugs and they tend to charge immense premiums. In most markets, the supply is somewhat proportionate to the demand, however in specific medical treatments, the target markets are very small, meaning small demand. Companies often depend on wide customer bases to distribute the cost of development. However, the market for Glybera, the most expensive gene therapy treatment to date, consists only of patients with lipoprotein lipase deficiency, limiting the target market to less than 10 people. Since the entire cost of developing Glybera is borne by only 10 people, the price tag is astronomical. In fact, it is probably safe to assume that the developer of Glybera still lost money charging $1 million per treatment.

One of the most outspoken defenders of deregulated pricing in the pharmaceutical industry is Martin Shkreli. Infamous for buying a one of one Wu-Tang album and his smug face, Shkreli perfectly exemplifies the dangers of deregulation. In most industries, absurdly high prices simply means that customers will find cheaper alternatives, but in the pharmaceutical industry, options are limited, giving all of the power to the select few companies that produce a certain drug. Martin Shkreli took advantage of this power when he raised the price of Daraprim, a life-saving immune-system drug used to treat parasitic infection as well as AIDS and Cancer patients, by 5,000%. He raised the price of the drug from $13.50 $750 for a single pill, leaving those who depended on the drug and many others outraged. His defense for the price increase was to fund future drugs that will better help the patients. He explained that the pharmaceutical company was not profitable at $13.50 a pill and that in order to grow the company, they had to become profitable to fund research and development. His defense makes sense from a business perspective, but there are many more factors to consider when determining the cost of a life saving medicine. If pharmaceutical companies are still loosing money charging $750 a pill and $1 million per treatment while countless people can’t afford the treatments they need, how can gene therapy and the pharmaceutical industry move forward and who will pay for it?


Dr. Mark McClellan, former FDA commissioner and current leader of the Duke-Margolis Center for Health Policy, organized a consortium at Duke to analyze gene therapy treatments and to brainstorm ways to help patients pay for treatments. Through their research, the consortium concluded that the healthcare system is far behind the medical industry and the advances it has made. Generally speaking, there are three parties involved in gene therapy, the patients seeking treatment, the companies developing and pricing the treatments, and insurance companies. It is in the patient’s best interest to pay as little as possible for medicine, it is in the company’s best interest to charge the patient enough to be profitable and to pay off the development of the drug, and it is the insurance company’s job to make sure they don’t pay for any of it. Since the three major parties involved have mutually exclusive interests, it is impossible for everyone to win. Another factor to consider is the fact that patients respond differently to the treatment, meaning there is a chance that a $1 million treatment is completely ineffective. McClellan explained what is essentially a refund policy for gene therapy treatments in which patients that do not experience any relief or remission of their disease within one month are entitled to a refund. That leaves many things open to interpretation such as the definition of relief or progress, and it still does not account for the possibility of a relapse outside of the refund-window. This makes things infinitely more challenging for not only the companies administering the treatments and the patients receiving them, but it also complicates the pricing structure for insurance companies. If a patient needs coverage for a $1 million dollar one-time treatment, they have to pay out an enormous sum all at once, the one thing that keeps insurance agents up at night.

With all of the factors working against the success of gene therapy, it is hard to predict what a successful implementation will look like on a grand scale. There are countless industries that are light-years ahead of their respective regulating entities. In medicine, that disconnect prevents patients from receiving what could be a life-altering, even life-saving procedures.

Despite the current odds, Nick Leschly, CEO of Bluebird Bio, re-assures us that he’s “confident we can figure it out because if someone has a very serious disease, and we can cure it, the system will find a way to reward that.”


The impact of Quantum Computers on Society


Similarly to our conversation on artificial intelligence, it’s safe to say that the first to fully create controllable quantum computers will be thousands of years ahead of his counterpart. One of the problems is that the very people that we do not want to have this type of power are the only ones who can afford the technology right now. Imagine the power that Google will have access to with the exponential growth that they are experiencing in the field of quantum computing. Match this with their advanced technology in the field of artificial intelligence and you have a match made in heaven. As our Machine Learning group has mentioned: we will have to face the fact that there will be a considerable number of jobs that will be replaced by artificial intelligence. To add to the issue, with the advance of quantum computing technology, this is a problem that we will have to address much earlier than we expected. If the only thing machines require to complete any given task is time to learn the task you have given it, which could be accelerated with the computational speed of a quantum computer, then how long will it take before there are machines working in hospitals operating with little to zero percentage error? Or even imagine if the power and speed of quantum computing was used to advance the field of gene therapy how fast could we engineer the perfect human that never dies?

The beneficial implications and uses of this technology are innumerable, but anything of such reward comes with a high level of risk. Longer life expectancy leads to overpopulation, which is a problem that we are not ready to face. We have also never faced any being of superior intelligence to us, which will be an issue of its own. We must make sure that this technology is placed in the right hands and is used for the beneficial advance of humanity. This has to be done appropriately the first time around because any slight error could threaten human existence as we know it.

Speaking of, Temporal Defense Systems recently purchased one of the most powerful quantum machines available and they will help build the basis for the security systems that will be necessary when this technology arrives on a larger scale in the near future. With quantum technology, they will be able to take security and defense to the next level with device to device authentication, increased speed of threat detection and real time security level rating. James Burrell, exclaims that there are considerable benefits associated with this technology, which are mainly their ability to solve more complex computational problems. This allows them to increase security on pace with the ever changing operational network.

As you may recall from previous posts, quantum technology works on an exponential scale. Because the basis of this technology is currently being made a reality and put into practice, progress will only rise exponentially from here. Remember all of the issues that we brushed off because they were too far ahead of their time? Well the reality is that these problems will be in our face before we know it and we must be prepared to take them on with viable solutions once they are here.

The Frightful Five and Gene Therapy

The latest developments within the gene therapy, medical community involves one of the Frightful Five. With Amazon recently purchasing 12 state pharmacy wholesale licenses, it is time to take a look at how the Frightful Five can make an impact on the healthcare, and specifically gene therapy sector, of the United States.

With a Frightful Five company such as Amazon at the brink of breaking into the healthcare industry, it could mean positive things for gene therapy. Already, pharmaceutical companies are looking to work with geneticists in researching cures to basically incurable diseases and with this interest from these companies, it can be inferred that the potential pharmaceutical companies see in gene therapy, is seen through the eyes of the Frightful Five as well. The contributions that these technological companies can make whether it be investment in research, as Google has done, or funding of drugs, therapies and clinical trials, these tech companies, although you do not want to trust with your health data, can contribute a lot to the gene therapy community.

Already we have seen how companies like Microsoft and Apple have taken steps to enter the health sector of the economy through their respective applications, NeXT, and Apple Health and there are no signs of them stopping there. With the Frightful Five taken initiative within the healthcare industry to fund gene therapy programs and rebuild the healthcare system of the nation, they can push bounds in the battle for updated health services. Society is no longer working in the typical, you get sick, you go to the doctor, model anymore. We are at the brink of preventing and curing disease to ensure it will never come back again, or to future generations and this is a business model that only a technological company can understand. Insurance and pharmaceutical companies are not prepared for the biotechnology being created by the likes of Apple and Google but these companies can ensure that they are. Soon both insurance and pharmaceutical companies alike will understand just how significant it is that they account for the digital technologies that will dominate the health market, or the digital technologies that will be made obsolete by gene therapy. The only way to prepare for such scenarios is by understanding the technologies and their capabilities, and in order to understand them, there needs to be a giant like Apple, Google, Amazon and Microsoft who can take these innovations to the mainstream.

In today’s society where new gene therapies are being approved by the FDA on what seems like a biweekly basis, it is important that this continues if not at a quicker rate but at a better quality rate. With the help of Apple and Google, the FDA already sees the potential in allowing these companies to use their health products to monitor people’s heart rates and more. Applying these physical technologies, given the hope that the data collected will be strictly monitored and controlled by the FDA in the utmost ethical manner, can prove crucial to bringing gene therapy to the mainstream. To be able to aide physicians and scientists in their diagnosis’ and research will only help generate a further interest into gene therapy and a better understanding of this sector of health overall.

There is, and should be, an outright fear of the Frightful Five collecting your health data. It is scary for a technological company to have this much control over your private and public life but in the end, the Frightful Five is so named because they have this control over everything and everyone. Since they do, the best way to get new innovations, progressions and more out there for people to actually see and use in their own lives, is to have the Frightful Five show it off. With Amazon’s purchasing of pharmacy licenses, they can change the business model of pharmacy to become more gene therapy based rather than drug based. With Apple and Google’s health devices, they can change the business model of the medical service industry from impersonal to personal. It does not feel comfortable to say that having these companies around is the best solution for us to bring reform to the gene and healthcare industries but for right now, these guys are all that we have to make sure this reform comes, and not in the future, but now.

The Pending Implications of Gene Therapy on Education

Despite the staggering number of applications for gene therapy that are currently being investigated, the list continues to grow almost by the day. MIT researchers have discovered a potential genetic link between ADHD and Autism, meaning that gene therapy may be helpful in curing if not improving care for both disorders. The research shows that there is potentially a relationship between the brain’s thalamic reticular nucleus (TRN), which blocks out sensory inputs that can be distracting, and both ADHD and Autism. Their research suggests that when the TRN activity is slowed, the brain has a harder time controlling distractions. This means that an adjustment to the TRN behavior could help control ADHD and Autism symptoms, as well as other attention-related disorders. The study is still in its infancy and the testing is being conducted on mice, but the results are very promising. The results suggest that a correction of one specific gene, Ptchd1, carried on the X Chromosome, can restore TRN functionality.

While the research is still in the early stages, the long-term implications of a successful TRN-correction procedure are huge. The CDC estimates that 11% of children ages 4-17, accounting for 6 million children, suffer from ADHD. That is 6 million students struggling to pay attention in classrooms, struggling to focus during exams, and ultimately struggling to find employment if the symptoms persist into adulthood. If a gene editing treatment is developed, there is a possibility that those children will be able to participate and even excel in the classroom in ways they could not have fathomed without the use of prescription medicine. By eliminating the need for drugs like Adderall and Ritalin, thus eliminating children’s dependency on them, the general health of those suffering from attention disorders will improve greatly. The unfortunate reality is that disorders like ADHD condition children to depend on medicine to function properly. It is not their fault because the medicine is necessary to focus and learn, that said, it is not healthy to develop a drug dependency at such a young age regardless of its purpose.

Gene therapy is already being applied to so many genetic disorders, with the possibility of curing ADHD and Autism, a new generation of smarter, faster, healthier, and stronger people seems inevitable. There will potentially be 6 million more students studying harder, retaining more information, and ultimately becoming better learners. If this trend is multiplied across generations, eliminating prohibitive, cognitive disorders in students, it could reshape the entire education system with many subsequent disruptions. There would potentially be less of a demand for remedial education and an increase in demand for higher education or a productive alternative. This will lead to a smarter overall population, a scenario that has countless positive implications. Once of which is increased economic stimulation, resulting from the inevitable increase in demand for employment. It may be a stretch, but it is possible that one genetic modification in 6 million students could transform the entire economy.

It is easy to become caught up in the seemingly infinite number of applications for gene therapy, but the price tag is certainly a reality check. The current price for Yescarta, a patient-specific gene therapy procedure designed to treat aggressive forms of blood cancer, is a whopping $373,000. At that price point, it makes far more sense to treat attention disorders such as ADHD with prescription drugs. However, as the technology evolves, the price will inevitably come down. We are in a strange transition period in many different industries and medicine is no exception. The new advancements being made are nothing short of groundbreaking, but it is no small task to bring a new procedure to the market. Due to FDA regulations, years of research and testing are required before the first human trials, and even then, successful implementation into the market is not guaranteed. In medicine, consumer demand for treatments is ahead of the technology, and the technology is way ahead of the FDA. This may slow down the implementation of new drugs and procedures, but their timelines do not detract in any way from the scope and severity of their implications.



Big Data in Genetics

Privacy is just not something to be expected in this day and age of technology and apart from the voluntary use of social media sites and applications that blatantly infringe on this right, people set themselves up for even more invasion when they request their genetic information. Ancestry and family lineage sites for the last few years has been picking up due to shows like “Who Do You Think You Are” and basically any tear-jerking story about long lost siblings who found one another through searching their lineage that you read on your Facebook page every morning. Lately, however, these sites, and not even getting to genetic testing sites like 23andMe, have been asking for more than just a last name and a few answers to personal questions. They have been asking for their customer’s DNA, their genetic data and the repercussions of allowing for a corporation to have the ultimate template and profile of you are greater than you even know. 

It seems harmless enough, just take a swab of your cheek and ship the sample to a company who within two weeks will mail you back a sheet that has your genetic lineage and maybe even a genetic profile to warn you of some genetic disorder you may have. Speaking on a positive note, what could this company have to gain from helping people learn more about their past and their own selves besides doing it from the perspective of a service to others. Well, in fact they have a lot to gain. Since so many people refrain from actually reading the privacy policy before shipping their genetic data through the mail, they do not even realize that they are signing away their right to sole ownership of the data that is collected. now has the rights to use the data that has been sent their way for their own purposes and if these purposes are to sell the information to the highest bidder, worst case scenario, they have the broad right to do so. Although legally companies such as and Helix do not have the license to sell away your genetic information to third parties, they do reserve the right, without your deliberate permission, or with it following your purchase of the genetic testing kit, to sell it to pharmaceutical and research companies. Now, this already seems like the only boundary these genetic testing sites are crossing is an ethical one where the whole hurt one for the greater good comes into play but when you find out how one of Ancestry’s partnerships is with Calico Labs, which is founded and operated by Google, Inc., you might sing a different tune. With a technology giant such as Google becoming so interested in the health sector of the nation’s economy, certain eyebrows should, and must be, raised before consenting to shipping off DNA that could potentially wind up in the hands to the very people who endlessly track and monitor its users. It is already a breach of rights when a genetic-information-collection company can claim rights to someone’s genetic information but when it can ultimately be shared with a corporation that is not always looking out for the welfare of its users, there needs to be a stop to it.

Before you decide to get your DNA tested for signs of genetic anomalies, keep one thing in mind, scientists are still working, to this day, trying to uncover the complex information of the millions of genes that reside in the human body. We simply do not know enough to give sufficient information to people who are just looking to see if they are carrying a genetic mutation. These tests cannot significantly provide life saving information and as a result, should be used with a grain of salt, or even less than that. Knowing this, as well as the fact that the genetic industry as a whole lacks substantial privacy laws and regulations, there should be more wariness when it comes to giving up your genetic data so easily. It seems unlikely for anyone to give up the password to their email but when shipping their DNA to an unknown lab somewhere in the United States, people do not seem to show the same concern for their privacy. Is this precaution for not sharing DNA samples a little extreme right now, considering how the healthcare and tech industry still cannot utilize genetic information for anything more than research? Perhaps. But, with millions of people having their data breached across the country, by more companies than one, how can we be sure that this genetic information will not be next? People need to expect more from their legislators when it comes to protecting their privacy so whether it be securing the pass-codes on their smartphones or protecting the use of their genetic data, people should care because even though it is not here yet, it is well on its way and preparation for it is vital.

The Future of Agriculture Depends on CRISPR

CRISPR does not only the capability of curing disease and altering the genes of human DNA, but it also has the potential of altering the genetic data of crops. GMOs have been a staple of the American farming industry for the better half of the last century because it produces efficiency within the economy. GMOs cut the time needed for crops to grow and for farmers and agricultural industries to turn a profit. Although it is beneficial to the economy, and trying to feed the nation as a whole, there are obvious repercussions for taking the easy way out.

These are the increased resistance to pesticides, the appearance of new allergies and other organism effects that are a result of consuming genetically modified foods. GMOs are spread primarily into crops through chemicals and other toxins that stimulate growth, help the crop retain its color and prevent the crop from being pest infested. What CRISPR can offer is the answer the organic farming community has been looking for: how can one ensure no food shortages, healthy options and increased human health? Although still a form of genetic modification, CRISPR does not come with the toxins that chemicals bring to people and the environment. The toxins that are emitted onto crops and harming the environment are creating more problems than solutions for the world. Instead, what genetically modifying tools can help create is altering the crop’s actual DNA and take away the harmful toxins that are in the environment and eaten by people altogether.

CRISPR is not only useful for altering the DNA of crops for farming purposes but for health purposes as well. This gene-editing tool can enable vegetables and fruits to carry even more health benefits and nutrients for those who cannot eat or drink certain foods.

As seen in the article linked above, corn now has the capability of carrying the same health benefits of meat and even more so, is on the verge of becoming the healthiest vegetable. Through doing this, imagine the hundreds of thousands of vegetarians who would no longer be deprived of the nutrients of meat or fish because those very same proteins are in the vegetables that they are more inclined to eat. If anything what CRISPR can accomplish is a win for the picky eaters who choose not to eat a certain food because they do not prefer the taste and now, they do not even have to. On that note, CRISPR has also been used to aid those with allergies such as those with allergies to gluten. Therefore, the allergies that people are suffering from, due to genetically modified chemicals, can now be combatted with gene therapy. As a result, these therapies can prove to be vital in easing the way of life for those with extensive food allergies whilst protecting people from future allergies to different crops because with all things remaining the same, not every human body will respond to a certain crop, genetically modified or not.

What CRISPR can also bring to the agriculture sector is a way to combat the environmental changes that are enveloping the world. With natural disasters and extreme weather becoming so common within the world, genetic engineering provides a solution to farmers and industries in this field. To think that CRISPR can be used to alter the genes of say oranges and allow for them to grow fruitfully in the winter or wheat that is capable of bracing extremely dry, or extremely wet, weather. This will make all types of crops accessible to places all over the world, battling hunger and providing for an a boost in the global economy for easily traded goods that be mass produced worldwide and shipped to various nations as a direct result. CRISPR gives nations the opportunity to expand their fight against world hunger in a way that was once thought to be impossible. Already this fall, apples that do not brown are going to be released into supermarkets and just with this launch alone, it should evident that genetically modifying the DNA of fruits and vegetables is not a thing of the future, but something that is happening and happening now.

Gene Therapy Update: How it Works and Why it’s Risky

Gene therapy has the potential to be the most innovative and disruptive medical procedure in the modern world, but it is still in its earliest stages of development. Technology moves faster than any other industry on the planet, but when paired with science and medicine, the pace may be so fast that it becomes detrimental. Gene therapy is equal parts technology and medicine and there will be an eternal struggle between the two. From a technological standpoint, it’s full steam ahead for companies like CRISPR. For those focused on the medical side however, there is a long road ahead. It is important to remember that the success of gene therapy depends first on the development of the technology, but without testing and a safe implementation in the medical field, no lives will be saved. It is much different than Silicon Valley pushing Face ID or a new app because in both of those instances, the faster the technology is available to consumers the better, or so they have us believe. When Apple pushes new tech like Face ID, they know that they can usually work out the bugs with a software update, but things aren’t that simple when the technology involves pumping new genes into people’s bodies.

The science behind gene therapy is just as amazing as the technology behind deciding which specific genes need attention. In order for a patient to receive the treatment, the new genes must be introduced into the body via a number of possible procedures. The most common of which include specifically placed injections or IV treatments, depending on the disease the treatment is aimed to fight and where the problem is within the body. Once inside the body however, the procedure is essentially the same. The new or modified genes are brought to their desired locations using a vector. The most common vectors are bacteria, viruses, and plasmids. They are the most common because while they are completely different, they all share the essential characteristic of being able to multiply quickly once in a cell. Under normal circumstances, their hyperactivity is a bad thing because it is the very reason that diseases are able to spread so quickly. In gene therapy however, this trait makes them the perfect candidates to transport genes. The video below provides a visual demonstration of this process during a treatment of a retinal disease.

The use of vectors is relatively consistent across all forms of gene therapy, however, there are several different methods used to treat different diseases. On the most basic level, there are two forms of gene therapy, somatic and germline. Somatic treatments are targeted at cells that do not produce eggs or sperm, therefore the treatment will not be passed down to subsequent generations. Conversely, germline treatments target cells that do produce sperm or eggs, which means that any alterations made to cells in the patient’s body will be passed down. Germline treatments are arguably more dangerous because if something goes wrong during the procedure or if the new genes are incorrect, the faulty DNA will be passed down until it is corrected by another procedure.

Under the umbrella of somatic and germline treatments are three main forms of therapies that vary depending on the problem. The therapies include gene augmentation, gene inhibition, and targeted cell attacks. Gene augmentation is used when a cell has faulty DNA that needs to be corrected. In this case, the new genes are attached to a vector that will reproduce in the desired cells, and will replace the existing DNA in the hopes of delivering the correct directions. Gene inhibition does the opposite, as gene inhibition treatments are designed to stop cells from behaving a certain way instead of correcting an undesired behavior. This form of gene therapy is useful for treating cancer patients because the goal is to slow down the reproduction of cancerous cells. This is achieved by attaching genes to a vector that will tell the cancerous cells to stop reproducing. Doctors also have the ability to target a cluster of cells that they wish to eliminate altogether. This is achieved by either injecting vectors that will kills the cells directly, or by using vectors that will trigger an immune system response which will in turn kill the desired cells. This is slightly less effective because the vectors are used to trick the body into fighting a disease that it did not otherwise recognize. Regardless of the objective of the various forms of gene therapy, vectors are the most effective vehicles to deliver new genes to cells.

While the vectors are very useful for delivering the new genes to their desired targets, there are many risks involved with the procedure. Success of the treatment aside, there are risks associated with the injection alone. It is a high-risk procedure because while it can be extremely effective, it can just as easily catalyze a series of detrimental reactions within the body. If the new DNA is delivered to the wrong cells, it can disrupt necessary functionality completely unrelated to the disease it was aimed to fight. If the vectors used in the procedure trigger an unexpected immune system response, the treatment can be rejected altogether or in some cases even cause organs to fail.

Jesse Gelsinger’s story serves as a grim reminder of the unintended consequences of gene therapy. Jesse suffered from a rare metabolic disorder called ornithine transcarbamylase (OTC) deficiency, which made him an ideal candidate for a gene therapy experiment at the University of Pennsylvania. The vector that was used in the procedure was a weakened cold virus which was delivered via injections. The doctors at Penn had tested their vector on mice, baboons, monkeys, and one other human patient, so they were confident that Jesse had a real chance of improving. What the doctors did not predict, was the overwhelming inflammatory response that started a chain reaction in Jesse’s body. A mere 24 hours after the injection, there was 11 times the normal amount of ammonia in Jesse’s blood, he was hyperventilating, his ears had swollen shut, he had developed a blood clotting disorder called jaundice, his kidneys were starting to fail along with his lungs, and his brain had started to shut down. Shortly after the doctors thought they had things under control, Jesse died. His death marked the first documented casualty resulting from gene therapy and left the doctors in shock.

Jesse’s premature death exemplifies the uncertainty involved with gene therapy, and there will undoubtedly be others. The technology is so new that is impossible to predict the outcome of the treatments with any degree of certainty. On paper, Jesse’s treatment should have worked but all of the years of testing and analysis that took place before the procedure were flipped upside down when his body started to reject the treatment. The best doctors in the world cannot explain what caused Jesse’s body to react the way it did which speaks to the unpredictable nature of the technology. Gene therapy is an amazing advancement for both technology and medicine, but it is still wildly imperfect and will require many more brave patients like Jesse before it becomes a dependable procedure.