biostudies-literatureUnknown32(2)Ellison SInnovate UKHematopoietic stem cell gene therapy (HSCGT) is a promising therapeutic strategy for the treatment of neurodegenerative, metabolic disorders. The approach involves the <i>ex vivo</i> introduction of a missing gene into patients' own stem cells via lentiviral-mediated transduction (TD). Once transplanted back into a fully conditioned patient, these genetically modified HSCs can repopulate the blood system and produce the functional protein, previously absent or non-functional in the patient, which can then cross-correct other affected cells in somatic organs and the central nervous system. We previously developed an HSCGT approach for the treatment of Mucopolysaccharidosis type II (MPSII) (Hunter syndrome), a debilitating pediatric lysosomal disorder caused by mutations in the iduronate-2-sMolecular therapy. Methods & clinical development101271https://www.ebi.ac.uk/biostudies/studies/S-EPMC11214401biostudies-literatureDesign and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy.PMC11214401Ellison SLearmonth YRoman-Rodriguez FJThrasher ABigger BWSmythe JBuckland KBonafont JBooth CHowe LBooth LKalra SHolley RDay VJones SfalseDesign and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy.Hematopoietic stem cell gene therapy (HSCGT) is a promising therapeutic strategy for the treatment of neurodegenerative, metabolic disorders. The approach involves the <i>ex vivo</i> introduction of a missing gene into patients' own stem cells via lentiviral-mediated transduction (TD). Once transplanted back into a fully conditioned patient, these genetically modified HSCs can repopulate the blood system and produce the functional protein, previously absent or non-functional in the patient, which can then cross-correct other affected cells in somatic organs and the central nervous system. We previously developed an HSCGT approach for the treatment of Mucopolysaccharidosis type II (MPSII) (Hunter syndrome), a debilitating pediatric lysosomal disorder caused by mutations in the iduronate-2-s2024-01-01T00:00:00Z2024 Jun2026-07-15T12:05:36.665Z2026-07-04T03:11:59.291ZS-EPMC112144013894693610.1016/j.omtm.2024.101271