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Jude Samulski

Showing results (181-190 of 198) with videos related to

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Journal of Virology|October 31, 2014
Enhanced transgene expression from recombinant single-stranded D-sequence-substituted adeno-associated virus vectors in human cell lines in vitro and in murine hepatocytes in vivoChen Ling, Yuan Wang, Yuan Lu, et al.
Human Molecular Genetics|November 16, 2014
Intermediate filament protein accumulation in motor neurons derived from giant axonal neuropathy iPSCs rescued by restoration of gigaxoninBethany L Johnson-Kerner, Faizzan S Ahmad, Alejandro Garcia Diaz, et al.
Molecular Therapy. Methods & Clinical Development|September 25, 2023
Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophyCaroline Le Guiner, Xiao Xiao, Thibaut Larcher, et al.
Nature Medicine|February 16, 2005
Remodeling of cortical bone allografts mediated by adherent rAAV-RANKL and VEGF gene therapyHiromu Ito, Mette Koefoed, Prarop Tiyapatanaputi, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|September 27, 2012
Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout miceKamal K E Gadalla, Mark E S Bailey, Rosemary C Spike, et al.
Human Gene Therapy|August 7, 2002
Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brainChristopher Janson, Scott McPhee, Larissa Bilaniuk, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 10, 2011
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vectorDawn E Bowles, Scott W J McPhee, Chengwen Li, et al.
Science Translational Medicine|December 21, 2012
Long-term follow-up after gene therapy for canavan diseasePaola Leone, David Shera, Scott W J McPhee, et al.
Human Gene Therapy|November 25, 2014
Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trialPaul E Monahan, Junjiang Sun, Tong Gui, et al.
The New England Journal of Medicine|October 8, 2010
Dystrophin immunity in Duchenne's muscular dystrophyJerry R Mendell, Katherine Campbell, Louise Rodino-Klapac, et al.
Pageof 20

Showing results (181-190 of 198) with videos related to

Sort By:
Pageof 20
Journal of Virology|October 31, 2014
Enhanced transgene expression from recombinant single-stranded D-sequence-substituted adeno-associated virus vectors in human cell lines in vitro and in murine hepatocytes in vivoChen Ling, Yuan Wang, Yuan Lu, et al.
Human Molecular Genetics|November 16, 2014
Intermediate filament protein accumulation in motor neurons derived from giant axonal neuropathy iPSCs rescued by restoration of gigaxoninBethany L Johnson-Kerner, Faizzan S Ahmad, Alejandro Garcia Diaz, et al.
Molecular Therapy. Methods & Clinical Development|September 25, 2023
Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophyCaroline Le Guiner, Xiao Xiao, Thibaut Larcher, et al.
Nature Medicine|February 16, 2005
Remodeling of cortical bone allografts mediated by adherent rAAV-RANKL and VEGF gene therapyHiromu Ito, Mette Koefoed, Prarop Tiyapatanaputi, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|September 27, 2012
Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout miceKamal K E Gadalla, Mark E S Bailey, Rosemary C Spike, et al.
Human Gene Therapy|August 7, 2002
Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brainChristopher Janson, Scott McPhee, Larissa Bilaniuk, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 10, 2011
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vectorDawn E Bowles, Scott W J McPhee, Chengwen Li, et al.
Science Translational Medicine|December 21, 2012
Long-term follow-up after gene therapy for canavan diseasePaola Leone, David Shera, Scott W J McPhee, et al.
Human Gene Therapy|November 25, 2014
Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trialPaul E Monahan, Junjiang Sun, Tong Gui, et al.
The New England Journal of Medicine|October 8, 2010
Dystrophin immunity in Duchenne's muscular dystrophyJerry R Mendell, Katherine Campbell, Louise Rodino-Klapac, et al.
Pageof 20