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Human Gene Therapy|May 20, 2026
Optimization of Alpha-1 Antitrypsin Expression from Adeno-Associated Virus VectorsQiushi Tang, Debora Pires-Ferreira, Christian Mueller, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 26, 2009
Modulation of exaggerated-IgE allergic responses by gene transfer-mediated antagonism of IL-13 and IL-17eChristian Mueller, Allison Keeler, Sofia Braag, et al.
Plos One|October 13, 2011
No immune responses by the expression of the yeast Ndi1 protein in ratsMathieu Marella, Byoung Boo Seo, Terence R Flotte, et al.
The Journal of Biological Chemistry|September 22, 2012
Histone deacetylase inhibitor (HDACi) suberoylanilide hydroxamic acid (SAHA)-mediated correction of α1-antitrypsin deficiencyMarion Bouchecareilh, Darren M Hutt, Patricia Szajner, et al.
American Journal of Respiratory Cell and Molecular Biology|August 21, 2010
Lack of cystic fibrosis transmembrane conductance regulator in CD3+ lymphocytes leads to aberrant cytokine secretion and hyperinflammatory adaptive immune responsesChristian Mueller, Sofia A Braag, Allison Keeler, et al.
Human Gene Therapy|March 23, 2007
Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sitesTerence R Flotte, Thomas J Conlon, Amy Poirier, et al.
Human Gene Therapy|December 7, 2007
Neonatal intraperitoneal or intravenous injections of recombinant adeno-associated virus type 8 transduce dorsal root ganglia and lower motor neuronsKevin D Foust, Amy Poirier, Christina A Pacak, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 6, 2025
Overcoming barriers to commercially pre-viable gene and cell therapies for rare and ultra-rare diseasesDavid Barrett, Paula M Cannon, Federico Mingozzi, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|September 17, 2002
A single-subunit NADH-quinone oxidoreductase renders resistance to mammalian nerve cells against complex I inhibitionByoung Boo Seo, Eiko Nakamaru-Ogiso, Terence R Flotte, et al.
Human Gene Therapy Methods|March 31, 2015
Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trialsAlisha M Gruntman, Lin Su, Qin Su, et al.
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