CRISPR Debate

A World Without Disease with Genetic Engineering

What if diseases such as Cystic Fibrosis and Huntington’s disease could be eradicated? A revolutionary bioengineering technique scientists have been working on over the past few years known as CRISPR Cas 9 could make this possibility a reality. CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, could very well cure some of the world’s most notorious inherited diseases, and possibly even cancer. CRISPR uses an enzyme called Cas9, a nuclease isolated from the bacteria Streptococcus pyogenes that can restore specific mutations by cutting target sequences of DNA. This bioengineering tool was first discovered when scientists noticed the bacteria that had segments of viral DNA. The enzyme Cas 9 stores pieces viral DNA as a blueprint or a copy, so that when the virus shows up again, the bacterium can recognize the invaders and instruct Cas 9 to kill the virus. Now, scientists have combined Cas 9 and guide RNA in order to target specific sequences in the human genome. One of the leading developers of the CRISPR system, Dr. Zeng Feng, claims this bioengineering tool can revert mutated disease-causing genes back to its original sequence. Acting like a pair of scissors, CRISPR can cut a particular target codon sequence that may remove inheritable diseases and/or conditions that affect not only the person being treated with CRISPR, but also their entire family line for generations to come. This scientific innovation could potentially create a world where these insufferable diseases no longer exist, and are just a part of history. However, this concept of human gene editing raises huge ethical controversy among Ethicists, which strain the progression of this new bioengineering system. Ethicists fear the uncertain effects CRISPR may have on future generations, as well as the social damage it could do if abused. This means CRISPR could be used for purposes other than medical treatment, like genetically altering babies for enhancement. However, CRISPR would be strictly regulated for medical purposes only, and used with caution. As of this month, the U.S. National Academy of Sciences say clinical trials for CRISPR may be permitted on human embryos when more research is done. The benefits may outweigh the risks though when considering all of the disease and suffering that could be eliminated for families affected. Although human gene editing does pose a potential risk if it was available to everyone, CRISPR would strictly be used as medical treatment for those who desire to free their family line from incurable genetic diseases and for patients who are on the brink of death waiting for an organ transplant. Human gene editing can improve and even save many lives.

The success CRISPR has cultivated in recent years have been astonishing safe and effective, from editing genes in plants for crop improvement to genes in live organisms. Scientists have even safely treated inherent conditions in animals, which serve as a model for human treatment of genetic disorders. Among these genetic conditions is Duchenne Muscular Dystrophy, which occurs when the codon for muscle growth is missing, also known as a nonsense mutation. In other words, since the whole gene codon is missing, it can not make the protein dystrophin. Without this protein, all muscles are weak, including the heart, and the person affected experiences frequent fatigue. In recent years, scientists have cured Duchenne Muscular Dystrophy in Mice using the Cas 9 enzyme . The affected mice were induced with the growth protein dystrophin, and over time, their muscles strengthened significantly and became well developed. The success in mice safely progresses the prospect of taking the next step and starting to use CRISPR on human embryo for testing and experimentation. Some may argue that just because it was successful in mice does not mean it is safe to use on human cells. Humans are much more intricate than mice and there can be unintentional alterations of genes other than the target gene if not careful. While this is a valid point, in 2015 scientists showed the world how safe CRISPR is editing live human cells by isolating HIV infected cells from patients, and were able to cut out the virus. Furthermore, just two months ago, Chinese researchers used the new technique to edit DNA in embryos from in vitro fertilization. If it is safe to use on living human cells, why not start treating conditions like Muscular Dystrophy, Cystic Fibrosis, and the degenerative Huntington’s disease, that have no other cure or alternative treatment? Why let people continue to suffer, or have parents burdened with the fact that they have a 50% chance of giving their condition to their child? Editing the human genome not only will help the individual (the unborn child) but the rest of the family line. Since CRISPR Cas 9 system holds this incredible power to completely wipe out detrimental disease or disorders from a whole family’s DNA, future generations will be affected drastically from just a single cut. Ethicists point out this may lead people to abusing CRISPR for social means, like genetically engineering their children to be tall, athletic, and intelligent. While these concerns consider the odds of social change, CRISPR would primarily, if not only, be used for medical purposes, and not for parents to use as specific design or enhancement. Most of the people who would use CRISPR suffer from disorders that will pass down to their children, and want rid them of the genetic condition.

Ethicists also point out that discrimination against those with already existing disabilities and conditions may occur. People with disorders and/or conditions may feel unwanted and ostracized if parents are cutting out and preventing conditions of their unborn children. Ethicists claim the parents will be granted with too much power and seem to be making a statement that all disorders and disabilities are horrible and can not lead good, fulfilled lives. It is true that conditions like blindness, hard of hearing, Down Syndrome, Autism, etc. are not bad at all, and people with them enjoy wholesome, happy lives. However, not all disabilities are the same. Those with Autism or Down Syndrome live completely different lives than those living with destructive conditions like Duchenne Muscular Dystrophy, or Huntington’s disease--A degenerative disease that effects of nerve cells--who must constantly rely on other people for survival. Day to day life is a struggle not having control over your own body movements, and not being able to do mundane tasks takes away the freedom every individual yearns to have. This doesn’t mean the individual can not be happy, but depending on the condition, life can be very challenging. Even if two people have the same disease, their perspectives will be different because they won’t have the same experiences. Conditions like blindness or Autism does have its hardships, but in order for the more fatal diseases like Cystic Fibrosis and Muscular Dystrophy, CRISPR must be available for those who suffer from these more detrimental conditions. Not every medical condition is bearable or has a cure/therapeutic treatment. All individuals with disabling conditions have unique experiences and endure different struggles.

Imagine you wake up, squinting up at your caretaker as she gently urges you to get up. Only you can’t exactly get up yourself. Nellie, your aid, pulls the white sheets back and scoops an arm under your legs. In response, you throw a heavy arm around her neck, and inhale deeply as Nellie transfers you to your chair. After brushing teeth, and using the bathroom, you sit at the dining table and wait for your scrambled eggs and sausage. The glorious smell is excruciating. You want to grab the pan out of Nessie’s grip and wolf down breakfast. Only you can’t. Your immobile arms and legs lie heavy at your sides as you are forced to patiently wait for Nessie to sit down and feed you. As Nessie finally stabs a small strip of scrambled egg and lifts it slowly to your mouth, you nearly rip the plastic fork from her hand. She scolds you in her usual tone, “Slow down now, you don’t want to choke!” You scoff and think to yourself I’m starved, can I just eat? All your focus is on is shoveling food in your mouth. Not only does Nessie scoop up microscopic pieces of food, it seems as though but the everyone and their mother is calling the house, prompting Nessie to put down the plate and run to answer. Staring at the now cold breakfast, You sigh as you hear Nessie argue yet again with the Pharmacy about medications not arriving on time. Although this may not happen to you, this is a normal part of life for those who have disabling conditions like Muscular Dystrophy or Cerebellar Palsy. My friend Nick, who lives with Cerebellar Palsy, experiences this all the time, if not every day.What if one day, Nick woke up and could get out of bed himself? What if Nick could do things independently, his own way? CRISPR has the opportunity to free those struggling with a debilitating condition that lacks autonomy and self sufficiency. This gene editing tool can enable people like Nick to be independent, something that healthy, able bodied people take for granted.

The CRISPR system can not only improve the lives of those with genetic diseases, but also has the ability to save thousands of lives. CRISPR can potentially save the many sick patients waiting for an organ transplant. In most organ transplants, the recipient’s immune system will reject the donor organ and start to attack it. On nearly every human cell is a surface protein referred to as the Major Histocompatibility Complex (MHC), which allow the immune system recognize itself and reject foreign tissues and/or organs. All human beings have a different enough MHC profile from one another that if one person’s cells is introduced in another person’s body, the recipient’s T cells will perceive them as foreign and react immediately. This immune response does not help the thousands of sick, immunocompromised people desperately waiting for a compatible donor with a matched MHC profile to get an organ. According to the U.S. Department of Health and Human Services, twenty two people die everyday waiting for an organ. Although this poses a major problem for many patients, CRISPR may be the solution. Scientists can use this bioengineering tool to create human organs in pigs that can then be transferred into patients who need an organ, without the issue of organ rejection. Within the past two years, scientists have modified over sixty genes in pigs that is an acceptable organ donor for humans by cutting out the parts the human immune system attacks. Physicians propose CRISPR can both edit the recipient’s T cells to suppress the response against donor organs, and edit the donor organ to shield it from the immune system’s programmed attack. With more research and more access to clinical testing, CRISPR could be used to help and save lives across the Country, and conquer major hurdle of tissue/organ transplantation that many doctors and scientists face today.

So far it seems as though CRISPR can help those who are already sick, whether it is because they are immunocompromised or have inherited disease from their parents. However, this bioengineering tool can help all people by fighting off one of the most notorious killers of the day, cancer. Cancer occurs when abnormal cells start growing out of control and eventually results in death. It is the second leading cause of death, killing one out of four Americans. Even those who consider themselves “healthy” and “able bodied” may get cancer sometime in the future. According to the National Cancer Institute, nearly 39.6% of men and women will be diagnosed with cancer at some point during their lifetimes. Cancer does not just affect sick people, but can affect all people, regardless of health or socioeconomic standing. Although cancer affects majority of the poor, it is still prevalent in all social classes and races across the nation. Fortunately, the Cas 9 enzyme can edit one’s immune (T ) cells in order for them to kill cancer cells more effectively. Since cancer cells actually hide from the immune system’s T cells, these T cells can be genetically modified as advanced cell hunters to kill cancer. Advancing the immune system could be a new therapy and treatment for cancer. With this potential innovation, modern cancer treatment like chemotherapy may not even be needed in the future. Cancer may even be wiped out with CRISPR editing and become a part of history.

Although there are many concerns about future society with gene editing, the powerful capability CRISPR has to wipe out genetic disorders surpasses all hypothetical concerns. Gene editing done on humans will essentially do more good than harm. The potential to rid human suffering caused by genetic conditions outweigh the social debate of how CRISPR will affect society. All innovations have and will continue to have some sort of impact of society, but this is for the health betterment of future generations. CRISPR should gain more clinical testing and research to advance this tool for the future world free of pain and suffering.