Delighted to have successfully defended my PhD last month!
We built DNA clearing HEK cells and designed AAV protein variants to study their function and improve AAV manufacturing!
Huge thanks to my PI @GenePlumber, everyone @UCLBiochemEng1, examiners @alanlparker & @pauldalby!
To understand hT7-AAV tropism, we performed a genome wide CRISPR screen on human primary T cells and excitingly, identified CD7, a T/NK cell marker, as a critical transduction factor. Huge effort @PL_Bernard@HuiCharlotte@gabbykimmerly
I am so excited to share our new paper in @Nature: the first programmable, site-specific integration of a large DNA payload into T cells in vivo.
A single IV injection results in therapeutic levels of TRAC-targeted CAR T cells in multiple models.
https://t.co/t3pyjHyGWS
a 🧵
Below is the story of the first patient treated with a prime-edited therapeutic, developed by @PrimeMedicine in a trial led by Dr. Élie Haddad and his team at CHU Sainte-Justine. This teenager suffered from chronic granulomatous disease (CGD), an immunodeficiency, and now—10 months after treatment—the patient is healthy, stable, and living with a functioning immune system. Tracy Attebury, whose story was previously told by @ginakolata@nytimes, was the second patient treated with a prime-edited therapeutic.
https://t.co/ZiVq1SmYsn
RFdiffusion3 now available! De novo protein design against any molecule
Try it on @tamarindbio today
RF3 shows success in designing de novo proteins against all-atom targets, including proteins, DNA and small molecules with diverse applications.
Today, we report a method for design of active enzymes, RFdiffusion2, in @naturemethods. For the first time, we are able to design enzymes with native-range catalytic activity.
We also are releasing our next frontier model, RFdiffusion3, code 👇
The first two lines of this paper illustrate quite how important this discovery is:
“Approximately half the worldwide population is at risk for dengue. No antiviral prophylaxis or treatment options are available.”
Excited to present the first major work after starting our lab at Stanford and the Arc this year: CRISPR-All, a unified genetic perturbation language for programming any major type of genetic perturbation simultaneously, in any combination, at genome scale, in human cells.
For visual learners like me, this is probably the best feature of Nano Banana Pro
I was reading a paper on Structural and thermodynamics stability of T4 Lysozyme mutants
A preprint out today from Arc Innovation Investigator Theo Roth, @AustinMHartman, @Oli_TN, and colleagues introduces CRISPR All–a unified programming language for editing human cells across all major genetic perturbation types at once.
🧬🤖 NEW PhD opportunity in my group: Using AI genome design tools to halt antimicrobial resistance spread!
Combining LLMs with synthetic biology to tackle one of medicine's biggest challenges! #SynBio#AMR#AI
https://t.co/NHJfDWKez4
Project application:
https://t.co/STuTruK4Yh
Dave Ricks has been at @EliLillyandCo for 20% of its 150-year history. He came to the pub, poured his own Guinness, and gave us a 2-hour state of the pharma union: drug prices, clinical trials, patent clocks, the rise of generics, Chinese peptides, compounding pharmacies, the US healthcare system, and how the broad success of GLP-1s have transformed Lilly's business. If you've never heard Dave speak before, you're in for a treat.
Timestamps:
00:00 Intro
05:08 Making R&D decisions
10:11 Clinical trials
24:59 Drug pricing
32:43 Stimulating more R&D
45:16 Pros and cons of US healthcare
58:20 New pharma business models
01:05:53 Stripe + enterprises
01:07:00 China
01:16:31 Generics
01:22:37 GLP-1s
1:37:43 r/Peptides
01:41:25 LillyDirect
01:46:35 Why do investors love LLY?
This is a fascinating interview – I've listened twice now, here's the 5 most interesting things
1. Inflammation drops fast on GLP1s (before weight loss) - one theory is that overeating itself may be inflammatory
Papers like this which demonstrate that alpha synuclein can act potently as prions, are exactly why you need to have proportionate safety measures in labs doing this kind of work. If you work on this and don’t have safety measures ask why! Great paper btw. https://t.co/qoRaVNLdVh
Today I had lunch with twelve young Irish people -- a wonderful bunch in their early 20s working on rockets, biotech, software/AI, etc. I enquired about whether any were planning on leaving Ireland over the next 3 years. All hands but one went up. I asked the holdout why he was staying; he clarified that he was also going to leave, just not in the next three years.
Mobility is a wonderful thing, but it feels worrisome if everyone in such a group concludes that they ought to emigrate.
This desire seems to generalize. A 2023 study suggested that more young people want to leave Ireland than any other EU country.
A grey day in Hamburg spent discussing all things virology and CRISPR screening at the DEFENDER symposium - an EU funded consortium on a mission to identify new targets and develop new therapies against high priority viruses.