@sebastien-levesque.bsky.social and I review therapeutic genome editing for inherited blood disorders rdcu.be/ewcZ3. We cover CRISPR, patient journey, DNA repair, patient access, genotoxicity, hemoglobinopathies, delivery, bone marrow failure, coselection, immunodeficiency, and more.

Excited to share our latest where we describe a gene editing method termed prime assembly that enables RNA programmable targeted integration of gene-sized DNA sequences in nondividing cells without dsDNA or nuclease-driven DSBs. See thread by incomparable @Seb. Also complementary work Sontheimer/Xue

In @nature.com we describe the use of scalable #proteinengineering & #machinelearning to predict millions of bespoke CRISPR enzymes, offering safer & more efficient genome editing tools 🧬🖥️ @rachelsilverstein9.bsky.social @mgbresearch.bsky.social @harvardmed.bsky.social www.nature.com/articles/s41...

New paper from my lab! Elevated mitochondrial membrane potential is a therapeutic vulnerability in Dnmt3a-mutant clonal hematopoiesis: rdcu.be/eh1Nb Complementary to new work from Steven Chan & @georgevassiliou.bsky.social as described by @goodell-lab.bsky.social: www.nature.com/articles/d41...

Francis Collins, the NIH Director for 12 years, led the Human Genome Project and other NIH efforts for 32 years, resigned today. Key words from his resignation letter www.nytimes.com/2025/03/01/u...