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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...
Αποθηκεύτηκε σε:
| Τόπος έκδοσης: | Expert Opin Orphan Drugs |
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| Κύριοι συγγραφείς: | , |
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
2015
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| Θέματα: | |
| Διαθέσιμο Online: | 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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