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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
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  Data: 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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  Data: <searchLink fieldCode="AU" term="%22Doshi+BS%22">Doshi BS</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Samelson-Jones+BJ%22">Samelson-Jones BJ</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Nichols+TC%22">Nichols TC</searchLink>; Department of Pathology and Laboratory Medicine and UNC Blood Research Center, University of North Carolina, Chapel Hill, NC 27516, USA.<br /><searchLink fieldCode="AU" term="%22Merricks+EP%22">Merricks EP</searchLink>; Department of Pathology and Laboratory Medicine and UNC Blood Research Center, University of North Carolina, Chapel Hill, NC 27516, USA.<br /><searchLink fieldCode="AU" term="%22Siner+JL%22">Siner JL</searchLink>; Divisions of Hematology and Medical Oncology, Department of Medicine, Washington University in St. Louis, St. Louis, MO 63110, USA.<br /><searchLink fieldCode="AU" term="%22French+RA%22">French RA</searchLink>; Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.<br /><searchLink fieldCode="AU" term="%22Lee+BJ%22">Lee BJ</searchLink>; Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.<br /><searchLink fieldCode="AU" term="%22Arruda+VR%22">Arruda VR</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Callan+MB%22">Callan MB</searchLink>; Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
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  Data: <searchLink fieldCode="JN" term="%22101624857%22">Molecular therapy. Methods & clinical development</searchLink> [Mol Ther Methods Clin Dev] 2024 Feb 02; Vol. 32 (1), pp. 101205. <i>Date of Electronic Publication: </i>2024 Feb 02 (<i>Print Publication: </i>2024).
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Cell+Press%22">Cell Press </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>101624857 <i>Publication Model: </i>eCollection <i>Cited Medium: </i>Print <i>ISSN: </i>2329-0501 (Print) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2223290501%22">23290501 </searchLink><i>NLM ISO Abbreviation: </i>Mol Ther Methods Clin Dev <i>Subsets: </i>PubMed not MEDLINE
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