@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.

CRISPR-based therapeutic genome editing for inherited blood disorders
Nature Reviews Drug Discovery - CRISPR-based technologies provide a diverse set of tools to correct pathogenic mutations. This Review describes the promise and the challenges of genome editing...