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ElMallah, Mai
One or more keywords matched the following items that are connected to ElMallah, Mai
Item TypeName
Concept Genetic Therapy
Academic Article Pompe disease gene therapy.
Academic Article Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9.
Academic Article Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice.
Academic Article Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model.
Academic Article Development of rAAV2-CFTR: History of the First rAAV Vector Product to be Used in Humans.
Academic Article Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease.
Academic Article AAV9 gene replacement therapy for respiratory insufficiency in very-long chain acyl-CoA dehydrogenase deficiency.
Academic Article Comparisons of Infant and Adult Mice Reveal Age Effects for Liver Depot Gene Therapy in Pompe Disease.
Academic Article Intralingual and Intrapleural AAV Gene Therapy Prolongs Survival in a SOD1 ALS Mouse Model.
Academic Article Intralingual Administration of AAVrh10-miRSOD1 Improves Respiratory But Not Swallowing Function in a Superoxide Dismutase-1 Mouse Model of Amyotrophic Lateral Sclerosis.
Academic Article Adeno-Associated Virus-Mediated Gene Therapy in the Mashlool, Atp1a3Mashl/+, Mouse Model of Alternating Hemiplegia of Childhood.
Academic Article What's new and what's next for gene therapy in Pompe disease?
Academic Article Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing.
Academic Article GAA replacement improves respiratory muscle, neural, and alveolar pathology in the pompe mouse.
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  • Gene therapy