Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A.

Fong, Sylvia; Yates, Bridget; Sihn, Choong-Ryoul; Mattis, Aras N; Mitchell, Nina; Liu, Su; Russell, Chris B; Kim, Benjamin; Lawal, Adebayo; Rangarajan, Savita; Lester, Will; Bunting, Stuart; Pierce, Glenn F; Pasi, K John; Wong, Wing Yen

Fong, Sylvia; Yates, Bridget; Sihn, Choong-Ryoul; Mattis, Aras N; Mitchell, Nina; Liu, Su; Russell, Chris B; Kim, Benjamin; Lawal, Adebayo; Rangarajan, Savita; Lester, Will; Bunting, Stuart; Pierce, Glenn F; Pasi, K John; Wong, Wing Yen.

Afiliación

Fong S; BioMarin Pharmaceutical, Novato, CA, USA. sfong@bmrn.com.
Yates B; BioMarin Pharmaceutical, Novato, CA, USA.
Sihn CR; BioMarin Pharmaceutical, Novato, CA, USA.
Mattis AN; Department of Pathology, University of California, San Francisco, San Francisco, CA, USA.
Mitchell N; Liver Center, University of California, San Francisco, San Francisco, CA, USA.
Liu S; BioMarin Pharmaceutical, Novato, CA, USA.
Russell CB; BioMarin Pharmaceutical, Novato, CA, USA.
Kim B; BioMarin Pharmaceutical, Novato, CA, USA.
Lawal A; BioMarin Pharmaceutical, Novato, CA, USA.
Rangarajan S; BioMarin Pharmaceutical, Novato, CA, USA.
Lester W; University Hospital Southampton, Southampton, UK.
Bunting S; University Hospitals Birmingham, Birmingham, UK.
Pierce GF; BioMarin Pharmaceutical, Novato, CA, USA.
Pasi KJ; Consultant, La Jolla, CA, USA.
Wong WY; Barts and the London School of Medicine and Dentistry, London, UK.

Nat Med ; 28(4): 789-797, 2022 04.

Article en En | MEDLINE | ID: mdl-35411075

Asunto(s)

Dependovirus; Hemofilia A; Dependovirus/genética; Dependovirus/metabolismo; Factor VIII/genética; Factor VIII/uso terapéutico; Terapia Genética/métodos; Vectores Genéticos/genética; Hemofilia A/genética; Hemofilia A/terapia; Humanos; ARN Mensajero; Transgenes/genética

Texto completo

Imprimir

XML

PubMed Links

Buscar en Google

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Dependovirus / Hemofilia A Tipo de estudio: Risk_factors_studies Límite: Humans Idioma: En Revista: Nat Med Asunto de la revista: BIOLOGIA MOLECULAR / MEDICINA Año: 2022 Tipo del documento: Article País de afiliación: Estados Unidos

Texto completo

Imprimir

XML

PubMed Links

Buscar en Google