Intralingual Administration of AAVrh10-miRSOD1 Improves Respiratory But Not Swallowing Function in a Superoxide Dismutase-1 Mouse Model of Amyotrophic Lateral Sclerosis

Lori A Lind, Ellyn M Andel,Angela L McCall,Justin S Dhindsa,Katherine A. Johnson,Olivia E. Stricklin,Christian Mueller,Mai K. ElMallah,Teresa E. Lever,Nicole L. Nichols

Human Gene Therapy（2020）

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摘要

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 moto...

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