Combinatorial ASO-mediated therapy with low dose SMN and the protective modifier Chp1 is not sufficient to ameliorate SMA pathology hallmarks

Combinatorial ASO-mediated therapy with low dose SMN and the protective modifier Chp1 is not sufficient to ameliorate SMA pathology hallmarks

Spinal muscular atrophy (SMA) is a devastating genetically inherited neuromuscular disorder characterized by the progressive loss of motor neurons in the spinal cord, leading to muscle atrophy and weakness. Although SMA is caused by homozygous mutations in SMN1, the disease severity is mainly determ...

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Bibliographic Details
Main Authors: A. Muinos-Bühl, R. Rombo, E. Janzen, K.K. Ling, K. Hupperich, F. Rigo, C.F. Bennett, B. Wirth
Format: Article
Language:English
Published: Elsevier 2022-09-01
Series:Neurobiology of Disease
Subjects:
Neuromuscular disorder
CHP1
SMA
SMN2
Antisense oligonucleotide
Genetic modifier
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996122001875

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http://www.sciencedirect.com/science/article/pii/S0969996122001875

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