UC Davis CRISPR Meeting Panel: A View from the Trenches on Human Disease

The second session at our CRISPR meeting was really powerful.

As with other posts from the UC Davis CRISPR meeting, since I was taking notes on the fly during this session, this post is a stream of bits from the different talks, often trying to capture the essence of key questions or ideas as the speakers talked so forgive the format.

Overall from this session, the potential future somatic-gene therapy kind of use of CRISPR for catastrophic, fatal diseases such as Huntington’s Disease is something that needs special attention and discussion. More data is needed to know how this will play out.

CRISPR meeting panel

Jacob Corn is a pioneering CRISPR scientist at IGI at Berkeley. I thought it was really cool that he passed a model of CRISPR-Cas9 around for audience. He made the analogy that what we are doing as a field is kind of like driving a car while still working on the car. It was striking when he pointed out the massive increase in publications on CRISPR-Cas9 going from 1 original paper in something like a year to start to now an average of 6 papers per day.CRISPR Model Jacob Corn

Kyle Fink. Kyle is a Huntington’s Disease (HD) researcher here at UC Davis, focusing on genetic approaches to HD including using CRISPR for somatic targeting. He gave an overview of HD and discussed targeting CAG repeats. A team could target the mutant HD DNA, RNA or the toxic protein itself. Targeting DNA could be more effective. Can we truncate the CAG repeats with CRISPR or other similar methods? Or could we silence the expression of the mutant allele specifically. We could use a combo of stem cell and gene therapy. I was impressed by the work he’s doing.

Vicki Wheelock. Dr. Wheelock is a UC Davis physician who cares for HD patients and also conducts research. She provided a very powerful perspective. HD manifests earlier than other neurodevelopmental diseases, typically in 30s and 40s at the peak of life. There are 30,000 cases in the US, but ripple effect affects a lot more people negatively. HD has many, diverse, and often tragic symptoms via the striatum being injured in HD. All treatments are now palliative. Stem cell therapies bring hope for replacement therapy such as MSCs with BDNF (which is taken out by HD). Another example is anti-sense oligo (ASO), which is the basis of a trial via spinal taps in Canada now. But ASO is not specific to mutant HD allele so need data on safety. She highlighted the huge unmet need. I learned something else new about HD in this talk in that HD families went underground during the eugenics movement. There is a stigma, but fortunately that is decreasing. Still a need for other new technologies such as CRISPR.

Judy Roberson. Judy is a wonderful patient advocate for the HD community for over 20 years and has served for 5 years on an FDA advisory panel. She was the first voting HD advocate on that panel. Part of the title of her talk was “HD: The Perfect First Model for CRISPR.” She gave a very moving talk.

She talked about how HD hit her family like a ton of bricks. Her MIL was affected in 30s. Took a long time for diagnosis and died at age 55. She and her husband Tim have 4 children. Tim’s brother Joe died of HD at 52. Judy’s husband was diagnosed at 39 and died in his early 50s. People with HD are often underground and there are worries about genetic discrimination. The stigma remains.

Judy talked about the unique HD program at UC Davis that has more than 500 patients. Some patients move here to be part of the program.

I was impressed with her guts and bluntness. For instance, she criticized the FDA for a number of reasons including lack of listening to patients and their overly conservative approach.

She also had some words for CIRM. “I love CIRM”, she said, but they urgently need some changes including more transparency and more of a role for patient advocates at each step. Judy wrapped up by invoking CRISPR as a “magic scissors” to tackle the mutant HD allele.

The panel also answered questions after they each spoke. For instance, I asked them about the potential use of CRISPR in human embryos to prevent HD.

Sweet 16 Science Twitter Accounts To Follow Innovative Medicine

STAT logoBelow are 16 Science Twitter accounts that I think are musts to follow for those interested in transformative science and also medicine from a wide range of diverse, thought-provoking perspectives.

I could list 160, but I’ve picked these 16 as a nice sampling with a lean towards those willing to take a risk in what they say or with unique views.

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Weekend science reads: lab labor, pubs, CRISPR shrooms, money & more

mushroom

Wikipedia image

Here are some weekend science reads for you.

A new type of “magic” mushroom? MIT Tech Review on lack of oversight of new GMO foods coming to your plate.

DrugMonkey’s piece on lab labor and specifically whether undergrads “count” drew a lot of comments there. Of course they count in my view.

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Weekend reads: sperm, stem cells, chimeras, STAP, DNA Apps

human sperm and eggHere are some cool science reads to enjoy this weekend.

Lab-grown sperm made from stem cells can’t swim, but are able to fertilize eggs, reports Eric Boodman over at STAT.

Mouse-human neural crest chimeras: Not only a matter of black and white. Clever title.

Antonio Regalado writes about a DNA App Store coming soon to a smartphone near you.

Jacob Corn has a new piece at the Washington Post entitled, CRISPR will change lives, but not only through genetic engineering.

Obokata tallies retraction number three, RetractionWatch reports.

Stem cells A guide to cellular transdifferentiation. I love direct reprogramming. 

Alzheimer’s and the Inflamed Brain: Their Links Run Deeper than Thought comes from Todd Dubnicoff over at CIRM’s The Stem Cellar.

Cool biomed blogs you may not heard of: a drugmonkey, a med student, #CRISPR, & more

Science blogging is somewhat of a communal exercise. At least, it should be and cool biomed blogs are a great community.

One of the most invigorating aspects of blogging is finding new blogs that are worthwhile and edgy.

The NodeBelow I list some of my recommendations for blogs that you might not be familiar with, but that you should definitely check out.

DrugMonkey Blog. A go-to source for perspectives and a reality check on NIH funding, careers, and sometimes a bit about drug science. Whoever DrugMonkey is, s/he is awesome. @drugmonkeyblog

Innovative Genomics Initiative (IGI) Blog. The IGI is Berkeley’s fantastic gene editing and genomics group that includes Jennifer Doudna and Jacob Corn. Jacob writes the IGI blog and his posts are definitely worth reading including many insights on CRISPR. @igisci 

The Node. Love stem cells and developmental biology? Then you’ll love The Node (symbol above), the timely web offering of news in this arena that feels very much like a blog from the folks who bring us the wonderful journal Development@the_Node 

Nerds Eye View Blog. UC Davis Medical Student Fiona Scott (pictured)Fiona Scott is funny and irreverent as she writes this edgy blog about medical school and more. Many of you will enjoy this site. @Nerdseyeview22

STAT. While STAT is not a blog, it is my vote for the best new biomedical science news outlet of 2015. It has a stellar group of science writers who also seem to have some of the best insider sources. You’ll often read about cool new developments here first. @statnews