Engineered hematopoietic stem and progenitor cells for mucopolysaccharidosis type I

INDICATION: Mucopolysaccharidosis

Hematopoietic stem and progenitor cells (HSPCs) engineered to overexpress IDUA could treat mucopolysaccharidosis I (MPS I; Hurler syndrome), a lysosomal storage disease caused by insufficient IDUA activity. The enzyme deficiency causes glycosaminoglycan accumulation and leads to progressive multi-organ dysfunction. In a genetic mouse model of MPS I, transplantation of HSPCs overexpressing IDUA reduced urine levels of glycosaminoglycans by 65%, normalized liver and spleen tissue levels of glycosaminoglycans, prevented dysplasia in the femur and skull zygomatic and parietal bones, and reduced impairments in locomotor activity and long-term memory...