CRISPR Ask Me Anything hits Reddit's front page

Using CRISPR to Treat Diseases

Dr. Doudna, along with program directors for the Centers of Excellence in Genomic Science (CEGS) Research Program in NHGRI's Division of Genome Sciences, answered nearly a hundred questions about CRISPR, the immune system, and how CRISPR can be used to treat specific diseases.

The hosts were Jennifer Doudna, Ph.D., professor of chemistry, biochemistry and molecular biology at the University of California, Berkeley, and members of her lab; Lisa Brooks, Ph.D., program director; Dan Gilchrist, Ph.D., program director; Mike Smith, Ph.D., program director; Lu Wang, Ph.D., program director; and Carolyn Hutter, Ph.D., acting division director, all in NHGRI's Division of Genome Sciences. Nicole Lockhart, Ph.D., program director, and Lawrence Brody, Ph.D., division director, both in NHGRI's Division of Genomics and Society, were there to tackle some of the ethics questions related to gene editing.

Here, we recap the event (or you can check out Reddit Science's page to read the full "AMA!")

From TellYouWhatitShwas

How concerning is the recently published finding that CRISPR-Cas9 faces antibody resistance in humans? Is there a good workaround?

From CRISPR Researchers

Hi, this is Kevin from the Innovative Genomics Institute. The recent finding of human immune responses to CRISPR-Cas9 proteins in human cells is an important finding. When gene therapies reached clinical trials in the late 1990's, unpredicted immune responses affected the patients. Possible work-arounds for CRISPR clinical trials would include using Cas9 proteins from different bacterial species, which humans bodies have never seen. This would include GeoCas9 from a thermophilic bacteria. Another work-around includes using ex vivo editing, in which the human cell is removed from the body, edited, and put back in the body. This is what researchers are testing out for Sickle Cell Disease.

The team received two questions from Congresswoman Lousie Slaughter (D, NY) about the challenges that woman face in science and inequity in access to life-saving technologies.

From Rep_Louise_SlaughterCongresswoman D-NY25

As the only microbiologist in Congress, I have long been a champion of women in STEM (science, technology, engineering, and mathematics). Dr. Doudna, you are a prominent scientist whose profile continues to rise as a direct result of your revolutionary scientific discoveries. What challenges and opportunities have you experienced as a woman in science?

Also, the discovery of CRISPR has brought the promises of using gene editing technology to cure debilitating and life-threatening diseases closer to reality, but with those promises come concerns that it could open new avenues for discrimination, inequality, and inequity. In 2008, I sponsored a bill that became law, called the Genetic Information Nondiscrimination Act or more commonly known as GINA. GINA prevents genetic discrimination in employment and health insurance. How do we ensure CRISPR-based genome editing doesn't create new avenues for genetic-based discrimination in future generations?

From CRISPR Researchers

Hi, this is Jennifer Doudna, and thanks for these questions. Challenges I've encountered include being told that "girls don't do science" (high school), doubting my abilities to do science (college) and trying to balance work and home life. Opportunities have included working with amazing scientists throughout my career, first as a student and later as a mentor. I feel very lucky to working on projects that involve science and broader questions about technology and society. And that leads to your second question about the potential for genetic discrimination. I think we need to work with scientists and stakeholders across the fields of medicine, agriculture and synthetic biology to ensure responsible progress with gene editing. For example, I'm working with a team at UC Berkeley and UCSF through the Innovative Genomics Institute to develop procedures for clinical use of gene editing in adults that will ultimately provide affordable options for patients with genetic disease.

Many folks had questions about the misunderstandings of gene-editing technology.

From th3spaceman

What would you say is the most misunderstood thing about CRISPR that can cause people to be opposed to your research? Also thank you for everything you guys have contributed to science, you're amazing!

From CRISPR Researchers

Hi, this is Carolyn Hutter from NHGRI - I think a major misunderstanding is that CRISPR is equivalent to germline editing (editing in egg or sperm cells) in humans, and that all work in this area should be opposed because of concerns related to genetic modification in humans. In fact, the vast majority of proposed applications of CRISPR are in basic research, as well as applications in plants, bacteria, and non-human animals. Further, a major focus of human applications are on somatic (non-inheritable) editing. A broader understanding of the potential benefits, risks and applications of CRISPR would likely lessen some of the opposition. For more about pubic opinions on gene editing go to: https://www.genome.gov/27569226/what-do-people-think-about-genome-editing//what-do-people-think-about-genome-editing/

Others wanted to know what educational materials exist for those wanting to design lessons for high school biology on CRISPR technology.

From TheseanSolution

I teach high school biology. Our standards are leaning more into molecular biology and there is room to incorporate more molecular biology labs into our curriculum. We talk about CRISPR and genetic engineering, but don't conduct labs associated with the concepts. Is the CRISPR technique likely to reach high school labs any time soon?

From CRISPR Researchers

Hi this is Kevin Doxzen from the Innovative Genomics Institute. We are working with two other non-profits to make a "CRISPR kit" specifically for high schools and educational settings. This kit will be affordable and come with a curriculum that meets state standards. We are hoping this will be available later in 2018. Please visit the Innovative Genomics Institute website (https://innovativegenomics.org/resources/educational-materials/genome-engineering/) for more educational material.

And lastly, how could CRISPR help with genetic diseases, and what more do we need to understand before that's a possibility?

From haechee

Is this technology something that could potentially be used to turn on/off genes in a living adult? If so, does that mean it could be used to combat the physical effects of PTSD/trauma that result in permanent genetic and hormonal changes? I realize this kind of thing is probably a ways off.

Thanks for what you do!

From CRISPR Researchers

Hi, Dan from NHGRI here. I think this is a great question. The possibility to use CRISPR-based technologies to turn genes on and off definitely exists. One thing to keep in mind - to use CRISPR to impact the course of any particular disease, we'd need to have a really strong understanding of the molecular basis of that disease, so we could predict what changes we'd need to make with CRISPR. Scientists are working hard to understand the biological underpinnings of PTSD and many other diseases, but there remains a huge amount to learn. We've got to keep hammering away to develop and apply techniques like CRISPR-Cas-based gene editing, but also to understand the underlying biology. As one example, NIH has started a new program developing quality tools for effective and safe genome editing of the disease-causing DNA within the non-reproductive ("somatic") cells (see here:https://commonfund.nih.gov/editing).