NIH R01 · 2024
In vivo hematopoietic stem cell genetic modification by viral particles
PROJECT SUMMARY Hematopoietic stem cell (HSC) gene therapies with lentiviral or CRISPR based gene modification are demonstrating favorable safety profiles and remarkable efficacy to treat severe monogenic blood disorders with unmet clinical need, including X-linked Severe Combined Immunodeficiency (X-SCID) and Sickle Cell Disease (SCD), with feasibility of rapid progress from target discovery and preclinical validation to first-in-human trials. Despite these successes, the technologies and procedures used in these trials require complex ex vivo bespoke manufacturing of autologous cell products, are expensive and difficult to scale to treat many patients, and intrinsically associated with…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.