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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...
Autores principales: | Payne, Julia G, Takahashi, Ayuko, Higgins, Michelle I, Porter, Emily L, Suki, Bela, Balazs, Alejandro, Wilson, Andrew A |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2016
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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 |
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