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Steven J Gray

Showing results (91-100 of 107) with videos related to

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Nature Reviews. Neurology|April 24, 2013
Progress in gene therapy for neurological disordersMichele Simonato, Jean Bennett, Nicholas M Boulis, et al.
JCI Insight|October 22, 2021
Dual-isoform hUBE3A gene transfer improves behavioral and seizure outcomes in Angelman syndrome model miceMatthew C Judson, Charles Shyng, Jeremy M Simon, et al.
F1000Research|April 23, 2015
A brief review of recent Charcot-Marie-Tooth research and prioritiesSean Ekins, Nadia K Litterman, Renée J G Arnold, 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 : 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.
Ebiomedicine|November 28, 2025
First-in-human high dose AAV9 intrathecal gene therapy for paediatric CLN7 disease: a phase 1, open-label, single ascending dose, non-randomised clinical trialBenjamin M Greenberg, Berge Minassian, Souad Messahel, et al.
Frontiers in Genetics|August 24, 2023
Long-term safety and dose escalation of intracerebroventricular CLN5 gene therapy in sheep supports clinical translation for CLN5 Batten diseaseNadia L Mitchell, Samantha J Murray, Martin P Wellby, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 26, 2025
Age-sensitive response of systemic AAV-mediated gene therapy in a newly characterized feline model of mucolipidosis IINettie K Pyne, Jessica Bagel, Charles Shyng, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 14, 2020
Current Clinical Applications of In Vivo Gene Therapy with AAVsJerry R Mendell, Samiah A Al-Zaidy, Louise R Rodino-Klapac, et al.
Nature Medicine|June 28, 2024
AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single patientJames J Dowling, Terry Pirovolakis, Keshini Devakandan, et al.
Pageof 11

Showing results (91-100 of 107) with videos related to

Sort By:
Pageof 11
Nature Reviews. Neurology|April 24, 2013
Progress in gene therapy for neurological disordersMichele Simonato, Jean Bennett, Nicholas M Boulis, et al.
JCI Insight|October 22, 2021
Dual-isoform hUBE3A gene transfer improves behavioral and seizure outcomes in Angelman syndrome model miceMatthew C Judson, Charles Shyng, Jeremy M Simon, et al.
F1000Research|April 23, 2015
A brief review of recent Charcot-Marie-Tooth research and prioritiesSean Ekins, Nadia K Litterman, Renée J G Arnold, 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 : 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.
Ebiomedicine|November 28, 2025
First-in-human high dose AAV9 intrathecal gene therapy for paediatric CLN7 disease: a phase 1, open-label, single ascending dose, non-randomised clinical trialBenjamin M Greenberg, Berge Minassian, Souad Messahel, et al.
Frontiers in Genetics|August 24, 2023
Long-term safety and dose escalation of intracerebroventricular CLN5 gene therapy in sheep supports clinical translation for CLN5 Batten diseaseNadia L Mitchell, Samantha J Murray, Martin P Wellby, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 26, 2025
Age-sensitive response of systemic AAV-mediated gene therapy in a newly characterized feline model of mucolipidosis IINettie K Pyne, Jessica Bagel, Charles Shyng, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 14, 2020
Current Clinical Applications of In Vivo Gene Therapy with AAVsJerry R Mendell, Samiah A Al-Zaidy, Louise R Rodino-Klapac, et al.
Nature Medicine|June 28, 2024
AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single patientJames J Dowling, Terry Pirovolakis, Keshini Devakandan, et al.
Pageof 11