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Molecular Therapy : the Journal of the American Society of Gene Therapy|October 23, 2017
Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral SeroreactivityNicole K Paulk, Katja Pekrun, Erhua Zhu, et al.Molecular Therapy. Methods & Clinical Development|February 2, 2019
Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar SyndromeFanny Collaud, Giulia Bortolussi, Laurence Guianvarc'h, 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.Science Translational Medicine|February 11, 2012
AAV2 gene therapy readministration in three adults with congenital blindnessJean Bennett, Manzar Ashtari, Jennifer Wellman, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|February 4, 2018
Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE ExpressionPatrice Vidal, Serena Pagliarani, Pasqualina Colella, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|June 24, 2014
Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primatesMarinee K Chuah, Inge Petrus, Pieter De Bleser, et al.Nature Communications|April 18, 2025
AAV vectors trigger DNA damage response-dependent pro-inflammatory signalling in human iPSC-derived CNS models and mouse brainHelena Costa-Verdera, Vasco Meneghini, Zachary Fitzpatrick, et al.Nature Medicine|June 3, 2020
IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodiesChristian Leborgne, Elena Barbon, Jeffrey M Alexander, et al.The New England Journal of Medicine|November 17, 2021
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia ALindsey A George, Paul E Monahan, M Elaine Eyster, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|December 3, 2009
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administrationFrancesca Simonelli, Albert M Maguire, Francesco Testa, et al.Pageof 14