{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["32(2)"],"submitter":["Ellison S"],"funding":["Innovate UK"],"pubmed_abstract":["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-s"],"journal":["Molecular therapy. Methods & clinical development"],"pagination":["101271"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC11214401"],"repository":["biostudies-literature"],"pubmed_title":["Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy."],"pmcid":["PMC11214401"],"pubmed_authors":["Ellison S","Learmonth Y","Roman-Rodriguez FJ","Thrasher A","Bigger BW","Smythe J","Buckland K","Bonafont J","Booth C","Howe L","Booth L","Kalra S","Holley R","Day V","Jones S"],"additional_accession":[]},"is_claimable":false,"name":"Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy.","description":"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-s","dates":{"release":"2024-01-01T00:00:00Z","publication":"2024 Jun","modification":"2026-07-15T12:05:36.665Z","creation":"2026-07-04T03:11:59.291Z"},"accession":"S-EPMC11214401","cross_references":{"pubmed":["38946936"],"doi":["10.1016/j.omtm.2024.101271"]}}