Zobrazeno 1 - 8
of 8
pro vyhledávání: '"Kenton T. Woodard"'
Autor:
John Paul Gaylor, Katharine J. Liang, Kenton T. Woodard, Mark A. Weaver, R. Jude Samulski, Ellen R. Weiss
Publikováno v:
Molecular Therapy. 25:765-779
NRF2 is a transcription factor that drives antioxidant gene expression in multiple organ systems. We hypothesized that Nrf2 overexpression could be therapeutically applied toward diseases in which redox homeostasis is disrupted. In this study, adeno-
Autor:
Richard Jude Samulski, Matthew L. Hirsch, Chengwen Li, M Wang, Kenton T. Woodard, A Crosby, Junjiang Sun
Publikováno v:
Gene therapy
Recent hemophilia B clinical trials using adeno-associated virus (AAV) gene delivery have demonstrated much lower coagulation factor IX (FIX) production in patients compared with the high levels observed in animal models and AAV capsid-specific cytot
Publikováno v:
Journal of Virology. 90:9878-9888
Many adeno-associated virus (AAV) serotypes efficiently transduce the retina when delivered to the subretinal space but show limited success when delivered to the vitreous due to the inner limiting membrane (ILM). Subretinal delivery of AAV serotype
Autor:
R. C. Spike, Paul D. Ross, Kenton T. Woodard, Sahana Nagabhushan Kalburgi, Mark E.S. Bailey, Stuart Cobb, Anne E. West, Kamal Ke Gadalla, R. Jude Samulski, Jie V. Deng, Steven J. Gray, Lavanya Bachaboina
Publikováno v:
Molecular Therapy. 21(1):18-30
Typical Rett syndrome (RTT) is a pediatric disorder caused by loss-of-function mutations in the methyl-CpG binding protein 2 (MECP2) gene. The demonstrated reversibility of RTT-like phenotypes in mice suggests that MECP2 gene replacement is a potenti
Autor:
Aravind Asokan, Telmo Llanga, Giridhar Murlidharan, Brian C. Gilger, Neil C Chungfat, Joanne Kurtzberg, R. Jude Samulski, William D. Bennett, Kenton T. Woodard, Matthew L. Hirsch, Melisa Vance
Publikováno v:
Scientific Reports
Although cord blood transplantation has significantly extended the lifespan of mucopolysaccharidosis type 1 (MPS1) patients, over 95% manifest cornea clouding with about 50% progressing to blindness. As corneal transplants are met with high rejection
Publikováno v:
Therapeutic Delivery. 1:517-534
This review aims to provide a broad overview of the targets, challenges and potential for gene therapy in the CNS, citing specific examples. There are a broad range of therapeutic targets, with very different requirements for a suitable viral vector.
Publikováno v:
Molecular Therapy. 24:S217-S218
AAV gene therapy has demonstrated success for the treatment of several ocular diseases with the tropism and efficiency of AAV retinal transduction being a function of the route of administration. Subretinal injection has been the primary route to del
Publikováno v:
Journal of morphology. 273(2)
Adult urodele amphibians possess extensive regenerative abilities, including lens, jaws, limbs, and tails. In this study, we examined the cellular events and time course of spinal cord regeneration in a species, Plethodon cinereus, that has the abili