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Recombinant adeno-associated virus vectors in the treatment of rare diseases

INTRODUCTION: An estimated 25 million Americans are living with rare diseases. Adeno-associated virus (AAV)-mediated gene therapy is an emerging therapeutic option for the more than 7,000 identified rare diseases. This paper highlights the benefits of AAV therapy compared to conventional small molec...

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Detalhes bibliográficos
Publicado no:Expert Opin Orphan Drugs
Main Authors: Hastie, Eric, Samulski, R. Jude
Formato: Artigo
Idioma:Inglês
Publicado em: 2015
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5034874/
https://ncbi.nlm.nih.gov/pubmed/27668135
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1517/21678707.2015.1039511
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