Loading...

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B

Muscle represents an important tissue target for adeno-associated viral (AAV) vector-mediated gene transfer of the factor IX (FIX) gene in hemophilia B (HB) subjects with advanced liver disease. Previous studies of direct intramuscular administration of an AAV-FIX vector in humans showed limited eff...

Fuld beskrivelse

Na minha lista:
Bibliografiske detaljer
Main Authors: Arruda, Valder R., Stedman, Hansell H., Haurigot, Virginia, Buchlis, George, Baila, Stefano, Favaro, Patricia, Chen, Yifeng, Franck, Helen G., Zhou, Shangzhen, Wright, J. Fraser, Couto, Linda B., Jiang, Haiyan, Pierce, Glenn F., Bellinger, Dwight A., Mingozzi, Federico, Nichols, Timothy C., High, Katherine A.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Hematology 2010
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2890180/
https://ncbi.nlm.nih.gov/pubmed/20335222
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2009-12-261156
Tags: Tilføj Tag
Ingen Tags, Vær først til at tagge denne postø!