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Pathogens & Immunity|April 18, 2019
Susceptibility to SIV Infection After Adenoviral Vaccination in a Low Dose Rhesus Macaque Challenge ModelIrene Bukh Brody, Roberto Calcedo, Mary J Connell, et al.
Nature Biotechnology|July 10, 2018
Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterolLili Wang, Jeff Smith, Camilo Breton, et al.
Human Gene Therapy. Clinical Development|June 25, 2013
Self-reactive CFTR T cells in humans: implications for gene therapyRoberto Calcedo, Uta Griesenbach, Daniel J Dorgan, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 15, 2017
5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin DeficiencyChristian Mueller, Gwladys Gernoux, Alisha M Gruntman, et al.
Human Gene Therapy. Clinical Development|March 23, 2016
Safety and Biodistribution Evaluation in CNGB3-Deficient Mice of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of AchromatopsiaGuo-jie Ye, Ewa Budzynski, Peter Sonnentag, et al.
Human Gene Therapy. Clinical Development|March 23, 2016
Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of AchromatopsiaGuo-jie Ye, Ewa Budzynski, Peter Sonnentag, et al.
Human Gene Therapy|May 26, 2011
Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim resultsTerence R Flotte, Bruce C Trapnell, Margaret Humphries, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 9, 2015
Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VIIBrittney L Gurda, Adrien De Guilhem De Lataillade, Peter Bell, 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.
Archives of Ophthalmology (Chicago, Ill. : 1960)|September 14, 2011
Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 yearsSamuel G Jacobson, Artur V Cideciyan, Ramakrishna Ratnakaram, et al.
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