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Molecular Therapy : the Journal of the American Society of Gene Therapy|June 16, 2025
Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe diseaseDavid D Fuller, Sabhya Rana, Prajwal P Thakre, et al.
Neurology. Genetics|November 17, 2021
Body Mass Index and Height in the Friedreich Ataxia Clinical Outcome Measures StudyMaya Patel, Ashley McCormick, Jaclyn Tamaroff, et al.
Molecular Therapy. Methods & Clinical Development|August 21, 2023
Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulationManuela Corti, Barry J Byrne, Dominic J Gessler, 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.
Molecular Therapy : the Journal of the American Society of Gene Therapy|May 7, 2025
Current clinical applications of AAV-mediated gene therapyBarry J Byrne, Kevin M Flanigan, Susan E Matesanz, 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.
Science Translational Medicine|January 4, 2023
Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophySharla M Birch, Michael W Lawlor, Thomas J Conlon, et al.
Plos One|March 18, 2025
Correction: A natural history study to track brain and spinal cord changes in individuals with Friedreich's ataxia: TRACK-FA study protocolNellie Georgiou-Karistianis, Louise A Corben, Kathrin Reetz, et al.
Annals of Neurology|March 22, 2025
Neuroimaging Biomarkers for Friedreich Ataxia: A Cross-Sectional Analysis of the TRACK-FA StudyNellie Georgiou-Karistianis, Louise A Corben, Eric F Lock, et al.
Plos One|November 21, 2022
A natural history study to track brain and spinal cord changes in individuals with Friedreich's ataxia: TRACK-FA study protocolNellie Georgiou-Karistianis, Louise A Corben, Kathrin Reetz, et al.
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