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A Penn Medicine and Children’s Hospital of Philadelphia (Children's Hospital) team will seek to develop treatments for three rare #genetic #diseases with the help of a $26 million grant from the National Institutes of Health. Kiran Musunuru spr.ly/6012Pw4Qr

🎉 Thrilled to share that our lab has received an NIH grant to develop CRISPR therapies for several rare diseases! I’m excited to contribute to the future of gene-editing therapies 🧬 alongside our colleagues at Children's Hospital

When I started working on #CRISPR to fight heart disease >10 years ago, I never imagined it might lead to cures for rare inborn errors of metabolism. Here’s how it’s happening! Presented/published today #ASHG23 #ASHG2023 ASHG plenary + AJHG + Human Genetics and Genomics Advances 🧵

That was such a lovely, clear, and well-laid out talk from Dominique L. Brooks - my favorite thus far! #ASHG2023

Wonderful start to the plenary session this evening by Dominique Brooks. #ASHG23 #ASHG2023

An exciting first step towards transformative therapies for our patients with rare inherited metabolic disease! @domi_niqueb, Madelynn Whittaker, Xiao Wang, Kiran Musunuru & Mohamad-Gabriel Alameh medicalxpress.com/news/2023-11-g… via Medical Xpress

It was a delight to present my new preprint at #CRISPRMED24! 🧬✂️ In bioRxiv, we report the utility of hybrid guide RNAs to improve the specificity and efficacy of base-editing therapies. See below for details! 🙌🏼

🏆Congratulations to the winners of the AJHG Award for Outstanding Trainee Publication; Dominique L. Brooks, BS, and Yosuke Tanigawa, PhD; and the Human Genetics and Genomics Advances Award for Outstanding Early Career Publication; Philip M. Boone, MD, PhD, and Gregory Costain, MD, PhD! 👏 #ASHG24