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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Udgivet i:Mol Ther
Main Authors: 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.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2017
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Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5542637/
https://ncbi.nlm.nih.gov/pubmed/28462816
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.04.003
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