Long-term lineage commitment in haematopoietic stem cell gene therapy.

ABSTRACT: Haematopoietic stem cell (HSC) gene therapy (GT) may provide lifelong reconstitution of the haematopoietic system with gene-corrected cells¹. However, the effects of underlying genetic diseases, replication stress and ageing on haematopoietic reconstitution and lineage specification remain unclear. In this study, we analysed haematopoietic reconstitution in 53 patients treated with lentiviral-HSC-GT for diverse conditions such as metachromatic leukodystrophy^2,3 (MLD), Wiskott-Aldrich syndrome^4,5 (WAS) and β-thalassaemia⁶ (β-Thal) over a follow-up period of up to 8 years, using vector integration sites as markers of clonal identity. We found that long-term haematopoietic reconstitution was supported by 770 to 35,000 active HSCs. Whereas 50% of tra

SUBMITTER: Calabria A 

PROVIDER: S-EPMC11618100 | biostudies-literature | 2024 Dec

REPOSITORIES: biostudies-literature