Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer

In previous work we transferred a human factor IX–encoding adeno-associated viral vector (AAV) into skeletal muscle of men with severe hemophilia B. Biopsy of injected muscle up to 1 year after vector injection showed evidence of gene transfer by Southern blot and of protein expression by IHC and im...

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Bibliografiska uppgifter
Huvudupphovsmän: Buchlis, George, Podsakoff, Gregory M., Radu, Antonetta, Hawk, Sarah M., Flake, Alan W., Mingozzi, Federico, High, Katherine A.
Materialtyp: Artigo
Språk:Inglês
Publicerad: American Society of Hematology 2012
Ämnen:
Gene Therapy
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC3321866/
https://ncbi.nlm.nih.gov/pubmed/22271447
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2011-09-382317
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