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

Article, Other literature type English OPEN
Mueller, Christian ; Tang, Qiushi ; Gruntman, Alisha ; Blomenkamp, Keith ; Teckman, Jeffery ; Song, Lina ; Zamore, Phillip D ; Flotte, Terence R (2012)
  • Publisher: Elsevier BV
  • Journal: Molecular Therapy, volume 20, issue 3, pages 590-600 (issn: 1525-0016, eissn: 1525-0024)
  • Related identifiers: doi: 10.1038/mt.2012.275, doi: 10.1038/mt.2011.292, pmc: PMC3293602, pmc: PMC3594022
  • Subject: Molecular Biology | Original Article | Genetics | Drug Discovery | Corrigendum | Pharmacology | Molecular Medicine
    mesheuropmc: congenital, hereditary, and neonatal diseases and abnormalities

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 seve... View more
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