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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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| Gepubliceerd in: | Expert Opin Orphan Drugs |
|---|---|
| Hoofdauteurs: | , |
| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
2015
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| Onderwerpen: | |
| Online toegang: | 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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