Gene therapy supports long-term reconstitution of patient hematopoietic stem cells in deficiency of adenosine deaminase 2

Résumé fourni par la source

, gene-corrected patient-derived HSPCs sustained long-term engraftment and multilineage reconstitution comparable to healthy controls. Integration site analysis confirmed a polyclonal pattern of hematopoietic reconstitution across all experimental settings, with no evidence of clonal dominance. Together, these findings demonstrate that ADA2 gene therapy with a LV is safe, maintains HSPC functionality, and enables durable hematopoietic reconstitution in a mouse model, providing a strong foundation for clinical translation and as a curative alternative to allogeneic HSCT in DADA2.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.