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Molecular Therapy. Nucleic Acids
|
March 20, 2026
An siRNA targeting <i>S6k1</i> identifies photoreceptor phospholipid metabolism as a contributor to lipid buildup in age-related macular degeneration
Shun-Yun Cheng, Delaney Giguere, San Kim, et al.
Neuroscience
|
November 7, 2016
AAV-mediated gene delivery attenuates neuroinflammation in feline Sandhoff disease
Allison M Bradbury, Tiffany A Peterson, Amanda L Gross, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
May 22, 2013
Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy
Allison M Bradbury, J Nicholas Cochran, Victoria J McCurdy, et al.
Science Translational Medicine
|
February 26, 2025
<i>BCKDHA-BCKDHB</i> digenic gene therapy restores metabolic homeostasis in two mouse models and a calf with classic maple syrup urine disease
Jiaming Wang, Laura E Poskitt, Jillian Gallagher, et al.
The New England Journal of Medicine
|
February 10, 2026
AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial
Connor J Lewis, Precilla D'Souza, Jean M Johnston, et al.
Medrxiv : the Preprint Server for Health Sciences
|
August 6, 2025
AAV9 Gene Therapy in GM1 Gangliosidosis Type II: A Phase 1/2 Trial
Connor J Lewis, Precilla D'Souza, Jean M Johnston, et al.
The Journal of Clinical Investigation
|
September 30, 2025
Five-year analysis of efficacy and safety of a bidirectional AAV gene therapy in Tay-Sachs sheep
Toloo Taghian, Jillian Gallagher, Stephanie Bertrand, et al.
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Search research articles
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Showing results (41-50 of 47) with videos related to
Sort By:
Page
of 5
You have reached the last page of results.
This site can display upto 47 results.
Molecular Therapy. Nucleic Acids
|
March 20, 2026
An siRNA targeting <i>S6k1</i> identifies photoreceptor phospholipid metabolism as a contributor to lipid buildup in age-related macular degeneration
Shun-Yun Cheng, Delaney Giguere, San Kim, et al.
Neuroscience
|
November 7, 2016
AAV-mediated gene delivery attenuates neuroinflammation in feline Sandhoff disease
Allison M Bradbury, Tiffany A Peterson, Amanda L Gross, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
May 22, 2013
Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy
Allison M Bradbury, J Nicholas Cochran, Victoria J McCurdy, et al.
Science Translational Medicine
|
February 26, 2025
<i>BCKDHA-BCKDHB</i> digenic gene therapy restores metabolic homeostasis in two mouse models and a calf with classic maple syrup urine disease
Jiaming Wang, Laura E Poskitt, Jillian Gallagher, et al.
The New England Journal of Medicine
|
February 10, 2026
AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial
Connor J Lewis, Precilla D'Souza, Jean M Johnston, et al.
Medrxiv : the Preprint Server for Health Sciences
|
August 6, 2025
AAV9 Gene Therapy in GM1 Gangliosidosis Type II: A Phase 1/2 Trial
Connor J Lewis, Precilla D'Souza, Jean M Johnston, et al.
The Journal of Clinical Investigation
|
September 30, 2025
Five-year analysis of efficacy and safety of a bidirectional AAV gene therapy in Tay-Sachs sheep
Toloo Taghian, Jillian Gallagher, Stephanie Bertrand, et al.
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of 5