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Neurobiology of Disease
|
November 22, 2020
Viral-based rodent and nonhuman primate models of multiple system atrophy: Fidelity to the human disease
David J Marmion, Angela A Rutkowski, Diptaman Chatterjee, et al.
Gene Therapy
|
December 22, 2023
CSTB gene replacement improves neuroinflammation, neurodegeneration and ataxia in murine type 1 progressive myoclonus epilepsy
Emrah Gumusgoz, Sahba Kasiri, Mayank Verma, et al.
Molecular Therapy. Methods & Clinical Development
|
May 13, 2017
Improved <i>MECP2</i> Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery
Sarah E Sinnett, Ralph D Hector, Kamal K E Gadalla, et al.
Human Gene Therapy
|
May 21, 2016
Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects GM2 Gangliosidosis in Sandhoff Mice
Karlaina J L Osmon, Evan Woodley, Patrick Thompson, et al.
Frontiers in Pharmacology
|
November 9, 2023
Efficacy of dual intracerebroventricular and intravitreal <i>CLN5</i> gene therapy in sheep prompts the first clinical trial to treat CLN5 Batten disease
Samantha J Murray, Martin P Wellby, Graham K Barrell, et al.
International Journal of Molecular Sciences
|
July 2, 2021
Investigating Immune Responses to the scAAV9-<i>HEXM</i> Gene Therapy Treatment in Tay-Sachs Disease and Sandhoff Disease Mouse Models
Shalini Kot, Subha Karumuthil-Melethil, Evan Woodley, et al.
Neurotherapeutics : the Journal of the American Society for Experimental Neurotherapeutics
|
March 13, 2026
Lafora disease gene therapy: EPM2A but not EPM2B overexpression results in Lafora body formation
Esther O Alao, Mehrnaz Sheibani, Jun Wu, et al.
Human Gene Therapy
|
May 21, 2016
Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease
Subha Karumuthil-Melethil, Sahana Nagabhushan Kalburgi, Patrick Thompson, et al.
Plos One
|
June 7, 2018
Sustained AAV9-mediated expression of a non-self protein in the CNS of non-human primates after immunomodulation
Arlene I Ramsingh, Steven J Gray, Andrew Reilly, et al.
Plos One
|
November 2, 2018
Correction: Sustained AAV9-mediated expression of a non-self protein in the CNS of non-human primates after immunomodulation
Arlene I Ramsingh, Steven J Gray, Andrew Reilly, et al.
Page
of 11
Search research articles
Search
Showing results (71-80 of 106) with videos related to
Sort By:
Page
of 11
Neurobiology of Disease
|
November 22, 2020
Viral-based rodent and nonhuman primate models of multiple system atrophy: Fidelity to the human disease
David J Marmion, Angela A Rutkowski, Diptaman Chatterjee, et al.
Gene Therapy
|
December 22, 2023
CSTB gene replacement improves neuroinflammation, neurodegeneration and ataxia in murine type 1 progressive myoclonus epilepsy
Emrah Gumusgoz, Sahba Kasiri, Mayank Verma, et al.
Molecular Therapy. Methods & Clinical Development
|
May 13, 2017
Improved <i>MECP2</i> Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery
Sarah E Sinnett, Ralph D Hector, Kamal K E Gadalla, et al.
Human Gene Therapy
|
May 21, 2016
Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects GM2 Gangliosidosis in Sandhoff Mice
Karlaina J L Osmon, Evan Woodley, Patrick Thompson, et al.
Frontiers in Pharmacology
|
November 9, 2023
Efficacy of dual intracerebroventricular and intravitreal <i>CLN5</i> gene therapy in sheep prompts the first clinical trial to treat CLN5 Batten disease
Samantha J Murray, Martin P Wellby, Graham K Barrell, et al.
International Journal of Molecular Sciences
|
July 2, 2021
Investigating Immune Responses to the scAAV9-<i>HEXM</i> Gene Therapy Treatment in Tay-Sachs Disease and Sandhoff Disease Mouse Models
Shalini Kot, Subha Karumuthil-Melethil, Evan Woodley, et al.
Neurotherapeutics : the Journal of the American Society for Experimental Neurotherapeutics
|
March 13, 2026
Lafora disease gene therapy: EPM2A but not EPM2B overexpression results in Lafora body formation
Esther O Alao, Mehrnaz Sheibani, Jun Wu, et al.
Human Gene Therapy
|
May 21, 2016
Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease
Subha Karumuthil-Melethil, Sahana Nagabhushan Kalburgi, Patrick Thompson, et al.
Plos One
|
June 7, 2018
Sustained AAV9-mediated expression of a non-self protein in the CNS of non-human primates after immunomodulation
Arlene I Ramsingh, Steven J Gray, Andrew Reilly, et al.
Plos One
|
November 2, 2018
Correction: Sustained AAV9-mediated expression of a non-self protein in the CNS of non-human primates after immunomodulation
Arlene I Ramsingh, Steven J Gray, Andrew Reilly, et al.
Page
of 11