Article
Intralingual Administration of AAVrh10-miRSOD1 Improves Respiratory But Not Swallowing Function in a Superoxide Dismutase-1 Mouse Model of Amyotrophic Lateral Sclerosis.
Human gene therapy - 1 Aug 2020
Lind Lori A, Andel Ellyn M, McCall Angela L, Dhindsa Justin S, Johnson Katherine A, Stricklin Olivia E, Mueller Christian, ElMallah Mai K, Lever Teresa E, Nichols Nicole L
Abstract excerpt
Amyotrophic lateral sclerosis (ALS) is a fatal disease characterized by degeneration of motor neurons and muscles, and death is usually a result of impaired respiratory function due to loss of motor neurons that control upper airway muscles and/or the diaphragm. Currently, no cure for ALS exists and treatments to date do not significantly improve respiratory or swallowing function. One cause of ALS is a mutation...
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