Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy.

Day, John W; Mendell, Jerry R; Burghes, Arthur H M; van Olden, Rudolf W; Adhikary, Rishi R; Dilly, Keith W

Day, John W; Mendell, Jerry R; Burghes, Arthur H M; van Olden, Rudolf W; Adhikary, Rishi R; Dilly, Keith W.

Afiliação

Day JW; Department of Neurology, Stanford University Medical Center, Stanford, CA, USA.
Mendell JR; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
Burghes AHM; Department of Pediatrics and Department of Neurology, The Ohio State University, Columbus, OH, USA.
van Olden RW; Department of Neurology and Department of Biological Chemistry and Pharmacology, The Ohio State University, Columbus, OH, USA.
Adhikary RR; Novartis Gene Therapies Switzerland GmbH, Rotkreuz, Switzerland.
Dilly KW; CONEXTS-Real World Evidence, Novartis Healthcare Private Limited, Hyderabad, India.

Mol Ther Methods Clin Dev ; 31: 101117, 2023 Dec 14.

Article em En | MEDLINE | ID: mdl-37822718

Palavras-chave

adeno-associated virus serotype 9; antibody titers; gene therapy; onasemnogene abeparvovec; retrospective analysis; seroprevalence; spinal muscular atrophy

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Observational_studies / Risk_factors_studies Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Estados Unidos

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