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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 15, 2013
Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease
Maria do Carmo Costa, Katiuska Luna-Cancalon, Svetlana Fischer, et al.
Science Translational Medicine
|
May 14, 2025
An AAV variant selected through NHP screens robustly transduces the brain and drives secreted protein expression in NHPs and mice
Luis Tecedor, Yong Hong Chen, David E Leib, et al.
Human Gene Therapy
|
January 6, 2017
Safety and Efficacy of Ex Vivo Donor Lung Adenoviral IL-10 Gene Therapy in a Large Animal Lung Transplant Survival Model
Tiago N Machuca, Marcelo Cypel, Riccardo Bonato, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 12, 2022
Evaluating the state of the science for adeno-associated virus integration: An integrated perspective
Denise E Sabatino, Frederic D Bushman, Randy J Chandler, et al.
Human Molecular Genetics
|
July 6, 2016
Transcriptome sequencing reveals aberrant alternative splicing in Huntington's disease
Lan Lin, Juw Won Park, Shyam Ramachandran, et al.
Biorxiv : the Preprint Server for Biology
|
August 30, 2023
APOE2 gene therapy reduces amyloid deposition, and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease
Rosemary J Jackson, Megan S Keiser, Jonah C Meltzer, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience
|
October 16, 2004
A mouse model of classical late-infantile neuronal ceroid lipofuscinosis based on targeted disruption of the CLN2 gene results in a loss of tripeptidyl-peptidase I activity and progressive neurodegeneration
David E Sleat, Jennifer A Wiseman, Mukarram El-Banna, et al.
Nature Medicine
|
October 19, 2021
Toxicity after AAV delivery of RNAi expression constructs into nonhuman primate brain
Megan S Keiser, Paul T Ranum, Carolyn M Yrigollen, et al.
Journal of Virology
|
July 24, 2009
Rho GTPases modulate entry of Ebola virus and vesicular stomatitis virus pseudotyped vectors
Kathrina Quinn, Melinda A Brindley, Melodie L Weller, et al.
Journal of Virology
|
August 21, 2002
In vivo gene transfer using a nonprimate lentiviral vector pseudotyped with Ross River Virus glycoproteins
Yubin Kang, Colleen S Stein, Jason A Heth, et al.
Page
of 19
Search research articles
Search
Showing results (161-170 of 187) with videos related to
Sort By:
Page
of 19
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 15, 2013
Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease
Maria do Carmo Costa, Katiuska Luna-Cancalon, Svetlana Fischer, et al.
Science Translational Medicine
|
May 14, 2025
An AAV variant selected through NHP screens robustly transduces the brain and drives secreted protein expression in NHPs and mice
Luis Tecedor, Yong Hong Chen, David E Leib, et al.
Human Gene Therapy
|
January 6, 2017
Safety and Efficacy of Ex Vivo Donor Lung Adenoviral IL-10 Gene Therapy in a Large Animal Lung Transplant Survival Model
Tiago N Machuca, Marcelo Cypel, Riccardo Bonato, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 12, 2022
Evaluating the state of the science for adeno-associated virus integration: An integrated perspective
Denise E Sabatino, Frederic D Bushman, Randy J Chandler, et al.
Human Molecular Genetics
|
July 6, 2016
Transcriptome sequencing reveals aberrant alternative splicing in Huntington's disease
Lan Lin, Juw Won Park, Shyam Ramachandran, et al.
Biorxiv : the Preprint Server for Biology
|
August 30, 2023
APOE2 gene therapy reduces amyloid deposition, and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease
Rosemary J Jackson, Megan S Keiser, Jonah C Meltzer, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience
|
October 16, 2004
A mouse model of classical late-infantile neuronal ceroid lipofuscinosis based on targeted disruption of the CLN2 gene results in a loss of tripeptidyl-peptidase I activity and progressive neurodegeneration
David E Sleat, Jennifer A Wiseman, Mukarram El-Banna, et al.
Nature Medicine
|
October 19, 2021
Toxicity after AAV delivery of RNAi expression constructs into nonhuman primate brain
Megan S Keiser, Paul T Ranum, Carolyn M Yrigollen, et al.
Journal of Virology
|
July 24, 2009
Rho GTPases modulate entry of Ebola virus and vesicular stomatitis virus pseudotyped vectors
Kathrina Quinn, Melinda A Brindley, Melodie L Weller, et al.
Journal of Virology
|
August 21, 2002
In vivo gene transfer using a nonprimate lentiviral vector pseudotyped with Ross River Virus glycoproteins
Yubin Kang, Colleen S Stein, Jason A Heth, et al.
Page
of 19