Lentiviral Vector-Mediated <i>Ex Vivo</i> Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis IVA Murine Model.

ABSTRACT: Mucopolysaccharidosis IVA (MPS IVA) is an autosomal recessive disease caused by a mutation in the N-acetylgalactosamine-6-sulfate-sulfatase (GALNS) gene resulting in progressive systemic skeletal dysplasia. There is currently no effective treatment available for this skeletal condition. Thus, the development of a new therapy stands as an unmet challenge in reversing or alleviating the progression of the disease. Our research, which could be a game-changer, hypothesizes that ex vivo lentiviral (LV) gene therapy (GT) could produce the supraphysiological level of active GALNS enzyme by hematopoietic stem cells (HSCs) transduced with LVs carrying the native GALNS gene under two different promoters (CBh and COL2A1), impacting bone and cartilage abnormalities in MPS IVA. We conditioned ne

SUBMITTER: Celik B 

PROVIDER: S-EPMC11693969 | biostudies-literature | 2024 Nov

REPOSITORIES: biostudies-literature