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Multilineage transduction of resident lung cells in vivo by AAV2/8 for α(1)-antitrypsin gene therapy

In vivo gene delivery has long represented an appealing potential treatment approach for monogenic diseases such as α(1)-antitrypsin deficiency (AATD) but has proven challenging to achieve in practice. Alternate pseudotyping of recombinant adeno-associated virus (AAV) vectors is producing vectors wi...

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Detalles Bibliográficos
Autores principales: Payne, Julia G, Takahashi, Ayuko, Higgins, Michelle I, Porter, Emily L, Suki, Bela, Balazs, Alejandro, Wilson, Andrew A
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4926859/
https://www.ncbi.nlm.nih.gov/pubmed/27408904
http://dx.doi.org/10.1038/mtm.2016.42