AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life.

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Title: AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life.
Authors: Doshi BS; Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.; Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Samelson-Jones BJ; Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.; Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Nichols TC; Department of Pathology and Laboratory Medicine and UNC Blood Research Center, University of North Carolina, Chapel Hill, NC 27516, USA., Merricks EP; Department of Pathology and Laboratory Medicine and UNC Blood Research Center, University of North Carolina, Chapel Hill, NC 27516, USA., Siner JL; Divisions of Hematology and Medical Oncology, Department of Medicine, Washington University in St. Louis, St. Louis, MO 63110, USA., French RA; Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Lee BJ; Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Arruda VR; Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.; Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Callan MB; Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2024 Feb 02; Vol. 32 (1), pp. 101205. Date of Electronic Publication: 2024 Feb 02 (Print Publication: 2024).
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2329-0501
DOI:10.1016/j.omtm.2024.101205