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Future of rAAV Gene Therapy: Platform for RNAi, Gene Editing, and Beyond

The use of recombinant adeno-associated viruses (rAAVs) ushered in a new millennium of gene transfer for therapeutic treatment of a number of conditions, including congenital blindness, hemophilia, and spinal muscular atrophy. rAAV vectors have remarkable staying power from a therapeutic standpoint,...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther
Main Authors: Valdmanis, Paul N., Kay, Mark A.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2017
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5399734/
https://ncbi.nlm.nih.gov/pubmed/28073291
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2016.171
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