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Article

Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Mar 2012

Mueller Christian, Tang Qiushi, Gruntman Alisha, Blomenkamp Keith, Teckman Jeffery, Song Lina, Zamore Phillip D, Flotte Terence R

Abstract excerpt

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-associated virus (rAAV) vectors that incorporate microRNA (miRNA) sequences targeting the AAT gene while also driving the...

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Identifiers

PMID: 22252449
DOI: 10.1038/mt.2011.292
PMCID: PMC3293602

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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