Recombinant Adeno-Associated Virus Vector Genomes Take the Form of Long-Lived, Transcriptionally Competent Episomes in Human Muscle

Gene augmentation therapy as a strategy to treat alpha-1 antitrypsin (AAT) deficiency has reached phase 2 clinical testing in humans. Sustained serum levels of AAT have been observed beyond one year after intramuscular administration of a recombinant adeno-associated virus (rAAV) vector expressing t...

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Gepubliceerd in:Hum Gene Ther
Hoofdauteurs: Schnepp, Bruce C., Chulay, Jeffrey D., Ye, Guo-Jie, Flotte, Terence R., Trapnell, Bruce C., Johnson, Philip R.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Mary Ann Liebert, Inc. 2016
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Research Articles
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5374867/
https://ncbi.nlm.nih.gov/pubmed/26650966
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2015.136
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