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Jacob Corn, the scientific director of the Innovative Genomics Initiative, is clearly having a good time.

He’s excited about the future of genome editing “from the perspective of a technology that solves biological problems and attacks disease.” A biophysicist/biochemist, Corn came to IGI from Genentech where he worked in pre-pipeline discovery, understanding how proteins go wrong in cellular signaling environments.  “Now, ” he says,  “we’re just doing basic research…how to foster turning this into some kind of therapeutic tool.”

One reason Corn is having such a good time is the open academic environment, the people involved in CRISPR research. “They’re really wonderful.” Standing at gene editing’s frontlines of innovation and exploration, he revels in the spirit of collaboration and collegiality among research gene editing scientists .  “It’s a fun field.. sharing,  collaborative… No one’s hiding their stuff…Everybody wants to be there first but meantime we’re still sharing reagents and sharing information on AddGene,  people just deposit their resources..everyone in the genome editing field has been putting this stuff up. That’s why it’s growing so fast”

Here’s the way sharing works.  Feng Zhang’s lab at MIT is a prolific source of plasmids created in the course of research.  One paper in Science, published shortly after the discovery of CRISPR/Cas9, attracted considerable attention.The Zhang Lab deposited the plasmids it created  in AddGene after which any researcher reading the paper could access those  specific plasmid vectors.

Innovative Genomics Initiative is a cooperative non-profit San Francisco Bay Area organization on the Berkeley Campus. Jennifer Doudna established IGI to foster collaboration among academic and industry researchers, to share and expand knowledge of CRISPR/Cas9 technology.  Labs at UC Berkeley, UC San Francisco and Stanford University are associated with IGI and its programs.

A good part of IGI’s mission is to share knowledge of molecular biology and  gene editing through outreach lectures and educational programming. “We love to teach,” says Corn.  A  major IGI event that looks like it might be an annual offering is the one week CRISPR workshop held on the Berkeley Campus.  This is a lecture based but practical hands-on program “that covers everything about CRISPR, including how to do it.”  Open to research scientists,  post docs and graduate students, this year’s program attracted several hundred applications by the submission deadline.

Earlier this year, AstraZeneca launched a research collaboration with IGI to identify gene targets relevant to cancer and other diseases and clarify their role in these conditions. This combination of IGI’s expertise in CRISPR gene editing and AstaZeneca’s is a collaboration designed to “positively impact drug discovery and development to hasten treatment to patients,” said Corn.

“The coming of CAs9 is start of this new era…  I’m interested in this technology because it’s cool and it allows us to address biological prblems that used to be off the table.  It lets us address   biological problems that used to be off the table.”

How long before CRISPR is used to treat patients?

“It depends on how you want to define that usem” says Corn. ” CRISPR is being used right now to refine CAR-Ts.

“There’s the first clinical application of true somatic gene editing and simple editing of a cell line that would be a good tool. Most companies I talk to are interested in CAR-Ts at the moment. They’ve been doing it for a while and it’s a total game changer in the oncology field. CRISPR genome editing is a way to make CAR-Ts even better. And going to the FDA for approvals shouldn’t be that hard since they’re already comfortable with CAR-Ts and gene editing is just making them better.

‘”Looking at a clinical trial of an edited somatic gene, that’s a longer game. We have a collaboration going with UCSF in Severe Combined Immune Deficiency (SCID) the ‘Bubble Boy’  disease where the patient has virtually no functioning immune system.

“Jennifer Puck the UCSF clinician on this was instrumental in having SCIDS testing mandated for newborns throughout California.  The earlier the intervention, the better. This is a genetic disease with a whole bunch of different genes involved.

“We’re interested in finding some of those variables  and eventually taking bone marrow, repair those genes and re-introducing them.  I hesitate putting a timeline on it but sooner rather than later…where sooner is years rather than months.

“The coming of CAs9 is start of this new era… I’m interested in this technology because it’s cool and it allows us to address biological problems that used to be off the table. ”
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