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The Journal of Clinical Investigation
|
March 23, 2023
Intrathecal AAV9/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies
Xin Chen, Thomas Dong, Yuhui Hu, et al.
Human Gene Therapy
|
April 12, 2011
Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors
Steven J Gray, Stacey B Foti, Joel W Schwartz, et al.
The Journal of Clinical Investigation
|
April 20, 2021
AAV9-mediated FIG4 delivery prolongs life span in Charcot-Marie-Tooth disease type 4J mouse model
Maximiliano Presa, Rachel M Bailey, Crystal Davis, et al.
Genes
|
August 26, 2023
SMRT Sequencing Enables High-Throughput Identification of Novel AAVs from Capsid Shuffling and Directed Evolution
Widler Casy, Irvin T Garza, Xin Chen, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
August 7, 2018
Longitudinal In Vivo Monitoring of the CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten Disease
Nadia L Mitchell, Katharina N Russell, Martin P Wellby, et al.
Neurobiology of Disease
|
October 9, 2016
N-acetylaspartate supports the energetic demands of developmental myelination via oligodendroglial aspartoacylase
Jeremy S Francis, Ireneusz Wojtas, Vladimir Markov, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience
|
April 24, 2015
Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophy
Jacqueline A Hawkins-Salsbury, Lauren Shea, Xuntian Jiang, et al.
Muscle & Nerve
|
August 29, 2025
T2 Hyperintensities in Gracile Tracts of Cervical Spinal Cord in Giant Axonal Neuropathy (GAN)
Diane Armao, Thomas W Bouldin, Diana X Bharucha-Goebel, et al.
Gene Therapy
|
February 1, 2025
Focused ultrasound widely broadens AAV-delivered Cas9 distribution and activity
Emrah Gumusgoz, Sahba Kasiri, Ibrahim Youssef, et al.
Nature Reviews. Neurology
|
April 24, 2013
Progress in gene therapy for neurological disorders
Michele Simonato, Jean Bennett, Nicholas M Boulis, et al.
Page
of 11
Search research articles
Search
Showing results (81-90 of 106) with videos related to
Sort By:
Page
of 11
The Journal of Clinical Investigation
|
March 23, 2023
Intrathecal AAV9/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies
Xin Chen, Thomas Dong, Yuhui Hu, et al.
Human Gene Therapy
|
April 12, 2011
Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors
Steven J Gray, Stacey B Foti, Joel W Schwartz, et al.
The Journal of Clinical Investigation
|
April 20, 2021
AAV9-mediated FIG4 delivery prolongs life span in Charcot-Marie-Tooth disease type 4J mouse model
Maximiliano Presa, Rachel M Bailey, Crystal Davis, et al.
Genes
|
August 26, 2023
SMRT Sequencing Enables High-Throughput Identification of Novel AAVs from Capsid Shuffling and Directed Evolution
Widler Casy, Irvin T Garza, Xin Chen, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
August 7, 2018
Longitudinal In Vivo Monitoring of the CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten Disease
Nadia L Mitchell, Katharina N Russell, Martin P Wellby, et al.
Neurobiology of Disease
|
October 9, 2016
N-acetylaspartate supports the energetic demands of developmental myelination via oligodendroglial aspartoacylase
Jeremy S Francis, Ireneusz Wojtas, Vladimir Markov, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience
|
April 24, 2015
Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophy
Jacqueline A Hawkins-Salsbury, Lauren Shea, Xuntian Jiang, et al.
Muscle & Nerve
|
August 29, 2025
T2 Hyperintensities in Gracile Tracts of Cervical Spinal Cord in Giant Axonal Neuropathy (GAN)
Diane Armao, Thomas W Bouldin, Diana X Bharucha-Goebel, et al.
Gene Therapy
|
February 1, 2025
Focused ultrasound widely broadens AAV-delivered Cas9 distribution and activity
Emrah Gumusgoz, Sahba Kasiri, Ibrahim Youssef, et al.
Nature Reviews. Neurology
|
April 24, 2013
Progress in gene therapy for neurological disorders
Michele Simonato, Jean Bennett, Nicholas M Boulis, et al.
Page
of 11