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Molecular Therapy : the Journal of the American Society of Gene Therapy|January 13, 2023
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapyMartin Schulz, Daniel I Levy, Christos J Petropoulos, et al.
Protein Engineering, Design & Selection : PEDS|January 23, 2025
Engineering of a lysosomal-targeted GAA enzymeNicholas Marze, Ilya Tikh, Susan Benard, et al.
Proceedings of the National Academy of Sciences of the United States of America|February 1, 2017
Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiencyRoberto Calcedo, Suryanarayan Somanathan, Qiuyue Qin, et al.
Human Gene Therapy|April 21, 2023
Complement Activation by Adeno-Associated Virus-Neutralizing Antibody ComplexesCara West, Joel D Federspiel, Kara Rogers, et al.
Proceedings of the National Academy of Sciences of the United States of America|April 30, 2003
Adeno-associated viruses undergo substantial evolution in primates during natural infectionsGuangping Gao, Mauricio R Alvira, Suryanarayan Somanathan, et al.
Molecular Therapy. Methods & Clinical Development|July 18, 2024
Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapiesAmit Chhabra, George Bashirians, Christos J Petropoulos, et al.
Human Gene Therapy. Clinical Development|September 28, 2013
Biodistribution of AAV8 vectors expressing human low-density lipoprotein receptor in a mouse model of homozygous familial hypercholesterolemiaShu-Jen Chen, Julio Sanmiguel, Martin Lock, et al.
Nature Medicine|July 18, 2006
Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsidLuk H Vandenberghe, Lili Wang, Suryanarayan Somanathan, et al.
Molecular Therapy. Methods & Clinical Development|March 7, 2022
<i>In vivo</i> overexpression of frataxin causes toxicity mediated by iron-sulfur cluster deficiencyClaudia Huichalaf, Tyler L Perfitt, Anna Kuperman, et al.
The Journal of Clinical Investigation|November 16, 2013
Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expressionChristian Mueller, Jeffrey D Chulay, Bruce C Trapnell, et al.
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