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

The safe correction of an inherited bleeding disorder in utero prior to the onset of organ damage is highly desirable. Here, we report long-term transgene expression over more than 6 years without toxicity following a single intrauterine gene transfer (IUGT) at 0.9G using recombinant adeno-associate...

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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5542637/
https://www.ncbi.nlm.nih.gov/pubmed/28462816
http://dx.doi.org/10.1016/j.ymthe.2017.04.003

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