Live Blogging #GeneEditSummit Day 1 Post #2: State of the Science, #CRISPR

Now we hear from the scientists on the front lines of CRISPR, covered in this post #2 of the Human Gene Editing Meeting. You can read Post #1 here.

Jennifer Doudna starts off the big human gene editing science session on the current state of the human gene editing science and CRISPR. She gave an overview of how this session will be organized including a panel discussion and questions from audience.

Maria Jasin, Memorial Sloan Kettering Cancer Center, was up next. She gave a nice overview of genome modification technology.

Emmanuelle Charpentier, Max Planck Institute of Infection Biology, spoke next. She explained the origins of CRISPR-Cas9. It is very cool to hear from one of the discoverers about how this technology came to be.

Up next is Jin-Soo Kim of Seoul National University / Institute for Basic Science. He talked about the concept of “Genome Surgery”. He talked also about the different kinds of off-target effects. “You cannot identify off-target effects of CRISPR in bulk populations of cells.”

Jonathan Weissman, University of California, San Francisco, talks about the use of CRISPR to control gene expression, a unique application. How do genes control complex organisms such as people? It is through elegant regulatory systems. Weissman uses the analogy of a piano with finite number of keys producing an infinite complexity of music.

J. Keith Joung, Massachusetts General Hospital and Harvard Medical School, talked about the work that his team is doing on CRISPR-Cas9. “A lot of improvement has been made in Cas9 systems in just the last few years”.

Next up Bill Skarnes from Wellcome Trust Sanger Institute gave some historical perspective on how hard gene editing used to be such as in mice. He highlighted the relative power of CRISPR systems to make different mouse models.

Feng Zhang, one of the top CRISPR-Cas9 innovators, spoke next. He shared his excitement about gene editing technology and to advance it to treat human diseases.

Update: Overall one striking thing about this session (so far as of 11:30am EST) is that the scientists are not voicing any societal, medical, or bioethical concerns. Perhaps this session was defined to be focused just on science.

Discussion and questions. Jennifer Doudna now follows up by asking some questions of the panelists.

• “What about prospects for controlling DNA damage?”
• “What do you see as the prospects for controlling genetic information (e.g. regulate expression)?”
  • Weissman–“challenge is delivery”.
  • Zhang –talks about both dead and active Cas9 for therapeutic options. Cas9 could be immunogenic and could be a challenge.

Question from audience on difference between CRISPR-Cas9 in the natural world and how it is being used by scientists.

Another question from audience: how can we get to a point where we feel it is safe from an off-target perspective? Zhang answer, “In part it will depend on the number of cells used. We need a standardized way to measure off-target effects”.

A third question–how long do you measure for changes or off-targets in cells? Joung answers this by saying that so far it has been arbitrary.

Another audience member, “What about regulations? Are you worried that some could be too restrictive?” Jasin, “I wish there were clearer discussions on ethical issues and guidelines”.