Gabriel M. C. Longo
139 posts
@genomewizard
PhD in Molecular Biology interested in genomics, DNA repair and gene editing. Research Fellow at HMS/MGH. Former @IMBmainz and @EMBL
Thursday morning, I had the privilege of speaking with the Muldoon family, whose 10-month-old son, baby KJ, was discharged Wednesday from @ChildrensPhila. Baby KJ was diagnosed with a rare urea cycle disorder and received a first-of-its-kind personalized CRISPR gene-editing therapy—customized specifically for his unique mutation. Thanks to the extraordinary expertise and compassion of the team at CHOP and the researchers who developed this pioneering treatment, baby KJ now has a second chance at life. The Muldoon family’s courage is nothing short of inspiring, and I offer my deepest gratitude to everyone at CHOP and beyond who made this medical milestone possible. This isn’t just a medical success story—it’s a powerful glimpse into the future of personalized medicine. I remain firmly committed to working with @NIHDirector_Jay, @DrMakaryFDA, @DrOzCMS, and others to ensure that: • HHS continues to invest in cutting-edge research that drives breakthrough therapies • Our regulatory frameworks keep pace with rapid innovation • No family is ever denied access to life-saving treatments because of cost Breakthroughs like this are why we do the work.
The genome-wide landscape of Cas-induced double-strand breaks and end structures is profiled at nucleotide resolution go.nature.com/44DO2Cc rdcu.be/ekKtv
