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Can we cure genetic diseases by rewriting DNA? | David R. Liu








In a story of scientific discovery, chemical biologist David R. Liu shares a breakthrough: his lab’s development of base editors that can rewrite DNA. This crucial step in genome editing takes the promise of CRISPR to the next level: if CRISPR proteins are molecular scissors, programmed to cut specific DNA sequences, then base editors are pencils, capable of directly rewriting one DNA letter into another. Learn more about how these molecular machines work — and their potential to treat or even cure genetic diseases.

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29 Comentários

  1. What an incredibly innovative approach to creating such a powerful and useful tool, thank you, Dr. Liu for taking the time to explain this very complex research in a digestible way. In my opinion, one of the most powerful things Dr. Liu said was about working with various professions to, “maximize the likelihood that base editing is applied thoughtfully, safely and ethically remains a critical obligation.” I couldn’t agree with this statement more. I believe the most important factor that will play into the future of genomic engineering innovations such as this one is the ethical implications. Since most of this speech was directed at the potential benefits of this research, I would like to briefly describe potential concerns that I have with these future innovations.

    It is incredibly important to countless children, families, and even adults that we work to find solutions to genetic diagnoses, however I strongly believe that we should approach the mechanisms for these solutions with caution. While the diseases Dr. Liu mentioned are all extremely severe and devastating, the ability to change these children’s genomes will likely lead to the question of which genetic abnormalities should be treated with this technology. For instance, autism spectrum disorder has a large genetic component. Should infants be screened for this disorder and subsequently treated? Autism spectrum disorder has a wide variety of associated medical conditions, so there are potentially good effects this technology could have on their health, however some of the greatest intellectuals in our history have been postulated to have this disorder such as Albert Einstein or even currently Elon Musk.

    If we start treating non-life-threatening genetic abnormalities, this, then leads to a slippery slope towards something described in medical ethics as “beyond therapy”: using medical therapies to improve upon someone’s genetics despite the lack of a serious medical condition. If, as in the autism spectrum disorder example, we are to begin treatment of intellectual disorders, parents may want to genetically screen their child for any and all potential challenges their child may face in their life, and as a society we could begin to lose the uniqueness of each human. As such, I wholeheartedly agree with Dr. Liu on the vital importance of collaboration of scientists, policymakers, and ethicists to begin to lay the groundwork for the future of genomic medicine. The distinction between what we consider a serious condition and what we consider as “beyond therapy” has many potential nuances and these distinctions have the potential to change healthcare and humanity in extremely impactful ways. It is for these reasons I believe that our society should begin to consider and plan for this future of medicine.

  2. The research and innovation in biochemistry performed by this scientist is truly remarkable. The adversity this lab overcame is astounding. However, I think an important question remains. Instead of can we cure genetic disease, I wonder if we should be asking the question should we cure genetic disease by rewriting DNA. At least, this will be a question as science advances into the future. The technological advances in medicine over the last 20 years have been incredible, life-changing, and lifesaving. I think everyone deserves to live a happy healthy life if this is their choosing, but those with genetic mutations do not necessarily get the choice. I believe the advancements with regards to base editing of the human genome is a great step in that direction. Possible issues with such capabilities of rewriting DNA poses the possibility of alternative use of such technology compared to the original intentions. Alternative use may embody using this technology to edit stem cells to give our offspring characteristics, traits that are desired such as height, strength, personality, intellectual ability, or appearance. So how do we prevent the alternative use of such technology in the future? Should we prevent the alternative use of such technology? These are questions I cannot answer other than possibly legislation. Regardless of the way in which the use of this technology is performed, it is hard to argue with the fact that humans would be interrupting evolution. Is this ethical? I believe the use of this base editing technology is ethical if it is used with the intentions of giving individuals with severely life-threatening conditions the opportunity do you live a long, happy, healthy life. If this technology is used to give individuals an advantage over other individuals, then I believe the use of this technology becomes unethical because at this point there is a violation of a biomedical principle known as justice. Distributive justice describes fairness in what people receive. If this technology were to become commercially used to give our offspring desirable traits, which likely would be restricted based on financial means, then there would exist an inequity. As a future physician, I take issue with this concept of use of medical technology. EW

  3. I used to have sickle cell anemia nd it would cause so many problems. It's good to know that there are cures being made for it because it's one of the lesser known diseases

  4. But I also think about reason behind this mutation. I think prevention is better than cure. Definitely they did incredible work but I think we should also think about the cause. Because of pollution , radiations and other factors genetic disease occurs . We are not only destroying our lives but also children's lives.

  5. I think it's really milestone in the history of genetic disease. 😊He and his team really did a great job ☺

  6. The work of David R. Liu and their team is an incredible feat of determination and dedication. Towards the end, it was mentioned the importance of using this technology ethically. Gene editing has proved to be controversial and ethically questioned due to the potential for abuse and expansion beyond the original intentions. How would we maintain the true nature of this technology, using it only to treat debilitating diseases? I believe there is a fine line between what can ethically be manipulated and what crosses over that line into performance-based preference. As health care professionals, we lead the implementation of this technology into our work. I am both eager to help cure illness and yet worried about the potentially consequences we may face as a society for interfering.

  7. Thank you so much. Am asad bashir, student of MPhil biotechnology from university of kotli azad kashmir. Your lecture helped me a lot to understand base editors.

  8. Very fascinating research and advancements into the field of genetics. It seems appropriate to use this technology to “fix” genetic mutations leading to diseases like he listed such as Progeria. This editing capability could lead to a tricky and convoluted path of knowing what type of genetic alterations are ethical or not. Is it ethical to decrease an individual's suffering? Is it ethical to enhance an individual's genome that will allow it to live a diseased free life? It is appropriate to reference the four ethical principles which are beneficence, non-maleficence, autonomy, and justice. Beneficence, non-maleficence, and autonomy are three of the medical ethical pillars that need to be addressed when discussing genetic alteration technology and advancements.

    Beneficence is the concept that physicians have a moral duty to provide care that is in the best interest of their patients. It could be argued that genetic alteration with the intent of alleviating disease is in the best interest of the patient. If gene editing is governed by an international ethical committee ensuring that the technology is used sparingly and only for the alleviation of disease, it does not violate the ethical pillar of beneficence.

    Non-maleficence is the obligation of a physician to do no harm. Genetic editing has the potential to disrupt normal embryologic development; thus, having hypothetical ability to harm a patient. If genetic alteration was conducted in a error-free way, then non-maleficence would not be violated.

    Lastly, autonomy is granting patient’s the right to make informed decisions about their medical care. On the first glance, one may think that autonomy is being violated in gene editing. The question is asked: how can one elect for genetic editing to occur if it is conducted during development and before birth? We must remember that minors do not have decision making capacity. The autonomy of a minor is limited due to their age. Is there difference in a parent electing for their infant to receive vaccinations and a parent electing for genetic editing to be performed?

    Genetic alteration is a field full of ethical considerations. It is imperative to consider the three concepts of beneficence, non-maleficence, and autonomy when pondering the ethical replications of genetic editing.

  9. Can doctor david liu help me by chance?

    I have been suffering with hot flashes and heat intolerance for 6 years now and so far nothing works. Could there be a way to treat it via altering my DNA ?? Im 40 now. However i had this issue as a baby and toddler also so i wonder if i have a genetic disposition to this 'ailment'. Please reach out to me if you can. Id really like to overcome this issue so i can exercise again and live a more active lifestyle.

  10. Hear me out… target SC with SICKLE CELL TRAIT.😮❤ Use SCT as the model for producing a healthy strand of hemoglobin. ?? Not sure if that’s an idea.

  11. God bless you DR OFENMU YOUTUBE CHANNEL you are really a true definition of a great man thanks for helping me get rid of genital herpes

  12. who else is here from there online bio class???????????????? hi i want to pass lol i hope i do. i think i will. i want ihop

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