In a landmark scientific feat published in @NEJM, a @ChildrensPhila & @PennMedicine team treated the first patient with personalized #genetherapy, designed uniquely for newborn KJ, who was diagnosed with a rare #ureacycledisorder at birth. Learn more: https://t.co/aUS67Sncdy
So proud of my PI @AhrensNicklas and close collaborator Dr. Kiran Musunuru for their work! Both are amazing mentors to their graduate students with a strong drive to help patients live better lives.
CHOP & @PennMedicine have delivered the first-ever personalized gene editing therapy for a patient with CPS1 deficiency, marking a major milestone in the application of CRISPR-based treatments.
Learn more about KJ & the future of personalized medicine: https://t.co/A9DTcNmxrR.
@ASGCTherapy 2024 is the most networking I've done in a long time! Loved presenting my work and project goals on CLN3 disease and seeing what others are achieving
Wrapping up #ncl2023 with @AhrensNicklas . Never have I ever experienced such an integrated group of scientists, willing to share so much preliminary work to better patient lives. #CLN#battens
What effect does climate change have on extreme weather events like droughts and wildfires? PWH Faculty Fellow @MichaelEMann addressed this question and others in a recent interview with @Penn_Today. Read here: https://t.co/962FkYENuw
Gene editing landscape
I pulled together 42 public & private companies and charted the preclinical & clinical assets of each, segmented by approach & editor
Some takeaways:
@hankgreen What are your opinions on/ can you do a public explanation on CAR-T (chimeric antigen receptor T) cells and similar therapeutics? - signed a gene therapy PhD student
If you like this, Give me a follow. I'm a PhD student in Gene Therapy also involved with Science Communication, Policy and Diplomacy! Sometimes hilarious.