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Molecular Therapy. Methods & Clinical Development|March 5, 2024
B cell focused transient immune suppression protocol for efficient AAV readministration to the liverJyoti Rana, Roland W Herzog, Maite Muñoz-Melero, et al.Human Gene Therapy. Clinical Development|November 22, 2017
Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe DiseaseManuela Corti, Cristina Liberati, Barbara K Smith, et al.Journal of Neuroengineering and Rehabilitation|March 10, 2026
Gait analysis reveals new outcome measures for monitoring disease progression in individuals with late-onset Pompe diseaseMireia Claramunt-Molet, Jordi Pegueroles, Ariadna Pi-Cervera, et al.Journal of Neuromuscular Diseases|March 4, 2025
Cardiopulmonary exercise testing as an integrative approach to explore physiological limitations in Duchenne muscular dystrophyMeghana Bomma, Donovan Lott, Sean Forbes, et al.Neurology|July 11, 2022
Natural History of Friedreich Ataxia: Heterogeneity of Neurologic Progression and Consequences for Clinical Trial DesignChristian Rummey, Louise A Corben, Martin Delatycki, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|December 14, 2020
Current Clinical Applications of In Vivo Gene Therapy with AAVsJerry R Mendell, Samiah A Al-Zaidy, Louise R Rodino-Klapac, et al.Annals of Neurology|October 17, 2020
Safety and Efficacy of Omaveloxolone in Friedreich Ataxia (MOXIe Study)David R Lynch, Melanie P Chin, Martin B Delatycki, et al.The Journal of Clinical Investigation|November 21, 2023
Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodiesStephanie M Salabarria, Manuela Corti, Kirsten E Coleman, et al.Translational Research : the Journal of Laboratory and Clinical Medicine|February 20, 2026
Mapping glycogen accumulation and treatment effect in Pompe disease with saturation transfer MRIQing Zeng, Yuguo Li, Derek Timm, et al.Biorxiv : the Preprint Server for Biology|January 7, 2025
Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe diseaseDavid D Fuller, Sabhya Rana, Prajwal Thakre, et al.Pageof 6