{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Harmatz P"],"funding":["NCATS NIH HHS","Sangamo Therapeutics"],"pagination":["3587-3600"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC9734078"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["30(12)"],"pubmed_abstract":["Zinc-finger nuclease (ZFN)-based in vivo genome editing is a novel treatment that can potentially provide lifelong protein replacement with single intravenous administration. Three first-in-human open-label ascending single-dose phase 1/2 studies were performed in parallel (starting November 2017) primarily to assess safety and tolerability of ZFN in vivo editing therapy in mucopolysaccharidosis I (MPS I) (n = 3), MPS II (n = 9), and hemophilia B (n = 1). Treatment was well tolerated with no serious treatment-related adverse events. At the 1e13 vg/kg dose, evidence of genome editing was detected through albumin-transgene fusion transcripts in liver for MPS II (n = 2) and MPS I (n = 1) subjects. The MPS I subject also had a transient increase in leukocyte iduronidase activity to the lower n"],"journal":["Molecular therapy : the journal of the American Society of Gene Therapy"],"pubmed_title":["First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B."],"pmcid":["PMC9734078"],"funding_grant_id":["UL1 TR002489"],"pubmed_authors":["Burton BK","Cao L","Rouy D","Harmatz P","Vaidya S","Meyer K","Villegas AG","Zeitler J","Kessler CM","Shiue LH","Muenzer J","Swenson W","Lau H","Miller W","Falaleeva M","Prada CE","Wong Po Foo C"],"additional_accession":[]},"is_claimable":false,"name":"First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B.","description":"Zinc-finger nuclease (ZFN)-based in vivo genome editing is a novel treatment that can potentially provide lifelong protein replacement with single intravenous administration. Three first-in-human open-label ascending single-dose phase 1/2 studies were performed in parallel (starting November 2017) primarily to assess safety and tolerability of ZFN in vivo editing therapy in mucopolysaccharidosis I (MPS I) (n = 3), MPS II (n = 9), and hemophilia B (n = 1). Treatment was well tolerated with no serious treatment-related adverse events. At the 1e13 vg/kg dose, evidence of genome editing was detected through albumin-transgene fusion transcripts in liver for MPS II (n = 2) and MPS I (n = 1) subjects. The MPS I subject also had a transient increase in leukocyte iduronidase activity to the lower n","dates":{"release":"2022-01-01T00:00:00Z","publication":"2022 Dec","modification":"2026-05-28T01:25:03.536Z","creation":"2025-06-01T02:33:49.229Z"},"accession":"S-EPMC9734078","cross_references":{"pubmed":["36299240"],"doi":["10.1016/j.ymthe.2022.10.010"]}}