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Adeno-associated viral vectors for the treatment of hemophilia

Gene transfer studies for the treatment of hemophilia began more than two decades ago. A large body of pre-clinical work evaluated a variety of vectors and target tissues, but by the start of the new millennium it became evident that adeno-associated viral (AAV)-mediated gene transfer to the liver h...

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Pubblicato in:Hum Mol Genet
Autori principali: High, Katherine A., Anguela, Xavier M.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Oxford University Press 2016
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4802375/
https://ncbi.nlm.nih.gov/pubmed/26614390
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddv475
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