Excited to share our discovery of a new programmable RNA-guided DNA-targeting system hiding inside bacteriophages that predates CRISPR.
We call it VIPR (Viral Interference Programmable Repeat), and it uses an entirely new logic to find its targets.
Thread + link below.
It just seems implausible this is what we are made of, essentially, nanotechnology about a billion years beyond anything we can design or make ourselves.
Today in @ScienceMagazine we report the development of a laboratory-evolved CRISPR-associated transposase (evoCAST) that supports therapeutically relevant levels of RNA-programmable gene insertion in human cells, a collaboration with @SternbergLab.
1/13
https://t.co/ZLHtZm5Gmw
Dual-function AAV vector to prevent alpha-1 antitrypsin deficiency
@muellerlab at UMass Medical School has developed a dual function approach using AAV #vectors targeting both #lung and #liver#diseases.
Find out more:
https://t.co/BdzuKCtEL7
To this day I get replies and DMs saying, “give up, you can’t end ALS.”
Let me make this simple:
I have ALS.
ALS is currently terminal.
It does not have to be terminal.
We’re on the precipice of change.
I will be part of making that change real and ending ALS.
Any questions?
A rewarding culmination of my 20 year academic career in #AAV#genetherapy ! I look forward to maturing what I incubated in the academic sector in the private sector to hopefully bring gene therapies to market and help patients with #rarediseases https://t.co/jVwNUs5Wqk
Muscle-Directed Delivery of an #AAV Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans: Molecular Therapy #GeneTherapy https://t.co/ylwAQsdrmQ
The @muellerlab at UMass Medical School has developed a #gene transfer approach that could be the future of ALS #treatment by silencing the #mutated SOD1 gene using microRNAs.
https://t.co/ABitKoOfHn
JONATHAN YOU CAN DO IT, WE CAN DO IT! Like and RT! Jonathan Maidment, a 24-year-old Alpha is about to finish his hike of the entire 2,650 miles of the Pacific Crest Trail. Help him get 2,650 LIKES on this pic to get a $20K donation towards his goal! #Hiking4ACure#Alpha1Awareness
Thanks to our Apic Bio team of riders and volunteers who participated in the annual Escape to the Cape Bike Trek 2019. Together we raised awareness for Alpha-1 and showed our dedication to #raredisease! #ridingforareason#curealpha1#Alpha1#GeneTherapy