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Methods in Molecular Biology (Clifton, N.J.)|May 19, 2011
Vector characterization methods for quality control testing of recombinant adeno-associated virusesJ Fraser Wright, Olga Zelenaia
Journal of Neurochemistry|December 1, 2004
Differential regulation of GLAST immunoreactivity and activity by protein kinase C: evidence for modification of amino and carboxyl terminiBala T S Susarla, Rebecca P Seal, Olga Zelenaia, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 23, 2008
Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responsesBernd Hauck, Samuel L Murphy, Peter H Smith, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 23, 2008
Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transferJeannette Bennicelli, John Fraser Wright, Andras Komaromy, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|June 28, 2012
Safe, long-term hepatic expression of anti-HCV shRNA in a nonhuman primate modelDavid A Suhy, Shih-Chu Kao, Tin Mao, et al.
The New England Journal of Medicine|December 7, 2017
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX VariantLindsey A George, Spencer K Sullivan, Adam Giermasz, et al.
The New England Journal of Medicine|April 29, 2008
Safety and efficacy of gene transfer for Leber's congenital amaurosisAlbert M Maguire, Francesca Simonelli, Eric A Pierce, et al.
Lancet (London, England)|October 27, 2009
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trialAlbert M Maguire, Katherine A High, Alberto Auricchio, et al.
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