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Orphanet Journal of Rare Diseases
|
February 3, 2019
Advancing the pathologic phenotype of giant axonal neuropathy: early involvement of the ocular lens
Diane Armao, Thomas W Bouldin, Rachel M Bailey, et al.
Current Protocols in Neuroscience
|
October 6, 2011
Production of recombinant adeno-associated viral vectors and use in in vitro and in vivo administration
Steven J Gray, Vivian W Choi, Aravind Asokan, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
November 13, 2020
Pre-clinical Gene Therapy with AAV9/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation
Xin Chen, Sarah Snanoudj-Verber, Laura Pollard, et al.
Molecular Therapy. Methods & Clinical Development
|
December 12, 2018
Efficacy of a Bicistronic Vector for Correction of Sandhoff Disease in a Mouse Model
Evan Woodley, Karlaina J L Osmon, Patrick Thompson, et al.
American Journal of Medical Genetics. Part A
|
December 5, 2024
SURF1 Deficiency: Expanding on Disease Phenotype and Assessing Disease Burden by Describing Clinical and Biochemical Phenotype
Saima Kayani, Victor Daescu, Hamza Dahshi, et al.
The Journal of Clinical Investigation
|
January 13, 2022
AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease
Xin Chen, Thomas Dong, Yuhui Hu, et al.
Trends in Neurosciences
|
February 3, 2016
Rett Syndrome: Crossing the Threshold to Clinical Translation
David M Katz, Adrian Bird, Monica Coenraads, et al.
Acta Neuropathologica Communications
|
June 17, 2017
Novel oligodendroglial alpha synuclein viral vector models of multiple system atrophy: studies in rodents and nonhuman primates
Ronald J Mandel, David J Marmion, Deniz Kirik, et al.
Plos One
|
March 14, 2013
In cellulo examination of a beta-alpha hybrid construct of beta-hexosaminidase A subunits, reported to interact with the GM2 activator protein and hydrolyze GM2 ganglioside
Incilay Sinici, Sayuri Yonekawa, Ilona Tkachyova, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
December 31, 2009
Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB)
Steven J Gray, Bonita L Blake, Hugh E Criswell, et al.
Page
of 11
Search research articles
Search
Showing results (41-50 of 106) with videos related to
Sort By:
Page
of 11
Orphanet Journal of Rare Diseases
|
February 3, 2019
Advancing the pathologic phenotype of giant axonal neuropathy: early involvement of the ocular lens
Diane Armao, Thomas W Bouldin, Rachel M Bailey, et al.
Current Protocols in Neuroscience
|
October 6, 2011
Production of recombinant adeno-associated viral vectors and use in in vitro and in vivo administration
Steven J Gray, Vivian W Choi, Aravind Asokan, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
November 13, 2020
Pre-clinical Gene Therapy with AAV9/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation
Xin Chen, Sarah Snanoudj-Verber, Laura Pollard, et al.
Molecular Therapy. Methods & Clinical Development
|
December 12, 2018
Efficacy of a Bicistronic Vector for Correction of Sandhoff Disease in a Mouse Model
Evan Woodley, Karlaina J L Osmon, Patrick Thompson, et al.
American Journal of Medical Genetics. Part A
|
December 5, 2024
SURF1 Deficiency: Expanding on Disease Phenotype and Assessing Disease Burden by Describing Clinical and Biochemical Phenotype
Saima Kayani, Victor Daescu, Hamza Dahshi, et al.
The Journal of Clinical Investigation
|
January 13, 2022
AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease
Xin Chen, Thomas Dong, Yuhui Hu, et al.
Trends in Neurosciences
|
February 3, 2016
Rett Syndrome: Crossing the Threshold to Clinical Translation
David M Katz, Adrian Bird, Monica Coenraads, et al.
Acta Neuropathologica Communications
|
June 17, 2017
Novel oligodendroglial alpha synuclein viral vector models of multiple system atrophy: studies in rodents and nonhuman primates
Ronald J Mandel, David J Marmion, Deniz Kirik, et al.
Plos One
|
March 14, 2013
In cellulo examination of a beta-alpha hybrid construct of beta-hexosaminidase A subunits, reported to interact with the GM2 activator protein and hydrolyze GM2 ganglioside
Incilay Sinici, Sayuri Yonekawa, Ilona Tkachyova, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
December 31, 2009
Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB)
Steven J Gray, Bonita L Blake, Hugh E Criswell, et al.
Page
of 11