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Heather Gray

Showing results (41-50 of 47) with videos related to

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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 degenerationShun-Yun Cheng, Delaney Giguere, San Kim, et al.
Neuroscience|November 7, 2016
AAV-mediated gene delivery attenuates neuroinflammation in feline Sandhoff diseaseAllison 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 therapyAllison 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 diseaseJiaming 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 TrialConnor 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 TrialConnor 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 sheepToloo Taghian, Jillian Gallagher, Stephanie Bertrand, et al.
Pageof 5

Showing results (41-50 of 47) with videos related to

Sort By:
Pageof 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 degenerationShun-Yun Cheng, Delaney Giguere, San Kim, et al.
Neuroscience|November 7, 2016
AAV-mediated gene delivery attenuates neuroinflammation in feline Sandhoff diseaseAllison 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 therapyAllison 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 diseaseJiaming 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 TrialConnor 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 TrialConnor 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 sheepToloo Taghian, Jillian Gallagher, Stephanie Bertrand, et al.
Pageof 5