Experts in gene editing, n=1 medicine development, in vivo delivery, rare diseases, and cell & animal models of genetic disorders have founded the Center for Therapeutic Genetics (CTG), a non-profit effort involving @broadinstitute@BostonChildrens@jacksonlab, as well as others from industry, academia, and patient advocacy organizations, to bring the promise of programmable medicines including base and prime editors to patients suffering from rare genetic diseases. This article from @ginakolata@nytimes frames the compelling need: https://t.co/OyrYCZtWIk
Official announcement: https://t.co/MwJCUarQnX
No words - only tears of joy. And bow of gratitude to Rebecca Ahrens-nicklas (CHOP) and Kiran Musunuru (Penn) and Sadik Kassim (Danaher) - our amazing partners in designing, derisking, and administering a CRISPR on demand to Baby KJ.
Today my friend and colleague Dr. Didem Demirbas and I are excited to present our team's research in Dr. @timyu's Lab on Personalized splice modulating ASO therapy for Zellweger Spectrum Disorder during Boston Children's Metabolism Grand Rounds! All are welcome to join via Zoom (you do not have to work at Boston Children's), would love to see you there!
Zoom link: https://t.co/W8jpQGPPVt
A few weeks ago I had the privilege of speaking at the annual meeting for @ALDConnect, the non-profit patient advocacy organization whose dedication to advancing health outcomes for patients with adrenoleukodystrophy is truly remarkable. I've been fortunate to meet and care for many ALD patients and their families in clinic with Dr. Eichler so this community has become very special to me. Thanks again for the opportunity to speak! I enjoyed getting to know all of the amazing patients, families, and researchers at the conference and I hope to come back next year.
https://t.co/s5mJYYcAjC
Very honored to receive an Emerging Generation Award from the American Society for Clinical Investigation @asci for early-career achievements and my research on organelles as I continue my NICU physician-scientist journey @UTSWnews@CRI_UTSW#neotwitter
https://t.co/WxidjFqwYA
The first rule of “Young Metabolic Club” is you have to talk about “Young Metabolic Club”!
The “Young Metabolic Society”: an interest group for young professionals in the field of metabolic medicine
Spread the word.
Brennenstuhl, Christ, & Kock
https://t.co/TVsDujWisd