As a cardiology consultant in the hospital, here’s my nomination for the heroes of the pandemic:
1. Nurses in the ER
2. Nurses in the ICU
3. Nurses on the floor
4. Nurses in the dialysis clinic
5. Nurses in the...
Proof-of-concept for an entire new way to treat heart attack:
a gene editing medicine,
administered once in life,
safely turn off a gene -->
permanently lower cholesterol or triglycerides
A ‘Cure for Heart Disease’? A Single Shot Succeeds in Monkeys
Amazing.
Once weekly injection, 30lbs average weight loss
Phenomenal results for a medicine to treat obesity
Once-Weekly Semaglutide in Adults with Overweight or Obesity | NEJM nejm.org/doi/full/10.10…
Can we precisely rewrite the genetic code - make a single DNA spelling change - in the liver of a human being for clinical effect?
YES!
Scientific milestone: VERVE-101 provides first proof-of-concept for in vivo DNA base editing in humans
Clinical implication: It’s early
Wow, say it with me:
“a functional cure” for sickle cell disease with CRISPR gene editing
All 31 patients with severe SCD, no vaso-occlusive crises (f/u 2 to 32 mos)
Similar durability and efficacy for beta-thal
👏🏽👏🏽👏🏽
@CRISPRTX@VertexPharmacrisprtx.gcs-web.com/news-releases/…
Does chronic disease need to equal chronic care? No
👇in @Nature from @VerveTx
One-time base editing Rx (in vivo liver),
durable⬇️LDL-C
In monkeys:
*⬇️90% PCSK9,⬇️60% LDL-C, now out to 10 mos
* VERVE-101: efficacy at LNP doses as low as 0.5 mg/kg
nature.com/articles/s4158…
Met with postdoc keen on staying in academia & applying for faculty jobs
I asked: "Have you put together a Description of Research Activities?"
👇🏽mine from April 2007 as I was just finishing my research training & applying for research faculty positions
Some thoughts...thread.
