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

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.

"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

Three things matter in #CRISPR therapies: delivery, delivery, and delivery.
Drs Alexandra Stanton, Pardis Sabeti, and colleagues expand our delivery toolbox with a novel set of AAV vectors active in nonhuman primates - bringing "CRISPR on the brain" to new meaning.
#womeninstem https://www.cell.com/med/fulltext/S2666-6340(22)00456-1

I'm thankful for the fact that @davidrliu and Alexis Komor and Nicole Gaudelli invented #CRISPR base editing that Eric Olson then used in great style to bring us closer to genome editing therapy for congenital heart disease.
https://www.science.org/doi/10.1126/scitranslmed.ade1633#