In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model.

Mattar, Citra N Z; Gil-Farina, Irene; Rosales, Cecilia; Johana, Nuryanti; Tan, Yvonne Yi Wan; McIntosh, Jenny; Kaeppel, Christine; Waddington, Simon N; Biswas, Arijit; Choolani, Mahesh; Schmidt, Manfred; Nathwani, Amit C; Chan, Jerry K Y

Mattar, Citra N Z; Gil-Farina, Irene; Rosales, Cecilia; Johana, Nuryanti; Tan, Yvonne Yi Wan; McIntosh, Jenny; Kaeppel, Christine; Waddington, Simon N; Biswas, Arijit; Choolani, Mahesh; Schmidt, Manfred; Nathwani, Amit C; Chan, Jerry K Y.

Afiliación

Mattar CNZ; Obstetrics & Gynaecology, Yong Loo Lin School of Medicine, National University of Singapore, Singapore 119077, Singapore. Electronic address: citramattar@nus.edu.sgp.
Gil-Farina I; Department of Translational Oncology, German Cancer Research Center and National Center for Tumor Diseases, 69120 Heidelberg, Germany.
Rosales C; UCL Cancer Institute, University College London, London WC1E 6BT, United Kingdom.
Johana N; Reproductive Medicine, K.K. Women's and Children's Hospital, Singapore 229899, Singapore.
Tan YYW; Reproductive Medicine, K.K. Women's and Children's Hospital, Singapore 229899, Singapore.
McIntosh J; UCL Cancer Institute, University College London, London WC1E 6BT, United Kingdom.
Kaeppel C; Department of Translational Oncology, German Cancer Research Center and National Center for Tumor Diseases, 69120 Heidelberg, Germany.
Waddington SN; Institute for Women's Health, University College London, London WC1E 6BT, United Kingdom; MRC Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg 2000, South Africa.
Biswas A; Obstetrics & Gynaecology, Yong Loo Lin School of Medicine, National University of Singapore, Singapore 119077, Singapore.
Choolani M; Obstetrics & Gynaecology, Yong Loo Lin School of Medicine, National University of Singapore, Singapore 119077, Singapore.
Schmidt M; Department of Translational Oncology, German Cancer Research Center and National Center for Tumor Diseases, 69120 Heidelberg, Germany.
Nathwani AC; UCL Cancer Institute, University College London, London WC1E 6BT, United Kingdom.
Chan JKY; Reproductive Medicine, K.K. Women's and Children's Hospital, Singapore 229899, Singapore; Duke-NUS Medical School, Singapore 169857, Singapore. Electronic address: jerrychan@nus.edu.sg.

Mol Ther ; 25(8): 1843-1853, 2017 08 02.

Article en En | MEDLINE | ID: mdl-28462816

Asunto(s)

Dependovirus/genética; Factor IX/genética; Expresión Génica; Técnicas de Transferencia de Gen; Vectores Genéticos/genética; Hemofilia B/sangre; Hemofilia B/genética; Animales; Anticuerpos Neutralizantes/inmunología; Anticuerpos Antivirales/sangre; Anticuerpos Antivirales/inmunología; Dependovirus/inmunología; Modelos Animales de Enfermedad; Femenino; Terapia Genética; Vectores Genéticos/administración & dosificación; Vectores Genéticos/efectos adversos; Hemofilia B/terapia; Humanos; Tolerancia Inmunológica; Hígado/metabolismo; Macaca fascicularis; Masculino; Embarazo; Factores de Tiempo; Transducción Genética; Transgenes

Palabras clave

adeno-associated viral vector; immune tolerance; intrauterine gene transfer; long-term expression; non-human primate; non-responder; vector integration

Texto completo

Imprimir

XML

PubMed Links

Buscar en Google

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Factor IX / Expresión Génica / Hemofilia B / Técnicas de Transferencia de Gen / Dependovirus / Vectores Genéticos Tipo de estudio: Prognostic_studies / Risk_factors_studies Límite: Animals / Female / Humans / Male / Pregnancy Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2017 Tipo del documento: Article

Texto completo

Imprimir

XML

PubMed Links

Buscar en Google