Sustained miRNA-mediated Knockdown of Mutant AAT With Simultaneous Augmentation of Wild-type AAT Has Minimal Effect on Global Liver miRNA Profiles

α-1 antitrypsin (AAT) deficiency can exhibit two pathologic states: a lung disease that is primarily due to the loss of AAT's antiprotease function, and a liver disease resulting from a toxic gain-of-function of the PiZ-AAT (Z-AAT) mutant protein. We have developed several recombinant adeno-ass...

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Detalles Bibliográficos
Autores principales: Mueller, Christian, Tang, Qiushi, Gruntman, Alisha, Blomenkamp, Keith, Teckman, Jeffery, Song, Lina, Zamore, Phillip D, Flotte, Terence R
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2012
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3293602/
https://www.ncbi.nlm.nih.gov/pubmed/22252449
http://dx.doi.org/10.1038/mt.2011.292

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3293602/
https://www.ncbi.nlm.nih.gov/pubmed/22252449
http://dx.doi.org/10.1038/mt.2011.292

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