Fyodor Urnov

466 Followers
43 Following
96 Posts
Genome editor @MCB UC Berkeley and the IGI. Beatles fanatic.
@jsherkow
My personal perspective is that - in the way childhood vaccines (MMR) have ultimately become near-universal because of a societal compact that it's a public good ... "CRISPR in a syringe" for certain classes of diseases should become same. The key difference w vaccines is you can use same one for 10m infants. W N=1 disease - you cannot.
Hence need for new framework.
@greally @gracebrowne
You're absolutely right in that a vertically integrated solution is the only way forward here.
I am embarrassingly ignorant of the "genomics for health" landscape in Ireland - I am curious whether these types of efforts (link below) could be a relevant example?
https://www.genomicsengland.co.uk/news/views-conditions-newborn-screening
Genomics England seeks views on choosing conditions for newborn…

The Newborn Genomes Programme is asking people to help it decide what principles should be used to choose conditions that newborns will be screened for.

Genomics England
A deeply affecting and inspiring read by Grace Browne @gracebrowne on a community affected by a severe genetic disease, and the clinicians+scientists behind a #CRISPR clinical trial for that disease by Intellia.
= the human face of #CRISPR impact on public health.
https://www.wired.co.uk/article/crispr-treatment-donegal-amy
Crispr’s Quest to Slay Donegal Amy

A trial using the gene-editing tool inside the body hints at treating, or even curing, a rare fatal disease—and it’s changing a community in the process.

WIRED UK
last boost (#LB?): LOVED Carolyn @bertozzi's answer when asked for whether her winning the Nobel Prize provides "an enhanced platform" to address gender issues in STEM (at time 22:00): "It's probably a question that's better posed to the men around the table...there are so many more of them and they are in a position to make change that's 9 times greater than my own position." 🙌🏽🙌🏽🙌🏽
Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing - Nature Biotechnology

Prime editing of large DNA sequences is achieved with two pegRNAs and site-specific recombinases.

Nature
The stream of letters (evoked by my NY Times piece) from families affected by severe N=1 genetic disease in my Inbox is devastating evidence that urgent reform is needed in how #CRISPR therapies are designed and delivered - and a huge motivator to make this reform happen.
This is from a letter sent by a father whose daughter is in a dire medical situation.
Every bit of technology - delivery, editor, etc - exists to build her a bespoke therapy.
Regulatory and lack of $ are in the way.
@jsherkow Absolutely yes. Tim and the N=1 collaborative are a guiding light for us.
"Unless things change dramatically, the millions of people #CRISPR could save will never benefit from it. We must, and we can, build a world with #CRISPR for all."
https://www.nytimes.com/2022/12/09/opinion/crispr-gene-editing-cures.html
Opinion | CRISPR Can Cure Disease by Editing a Person’s DNA. Now What?

Revolutionized medicine may be at hand, but barriers remain.

The New York Times
@ManiMota John would have enjoyed this greatly. it's his kind of humor.
@jsherkow Is this on the Stanford campus?
In their defense, they do have a remarkable Rodin collection and I guess felt obliged to diminish its impact using this?