The MuSK agonist antibody protects the neuromuscular junction and extends the lifespan in C9orf72-ALS mice

The disassembly of the neuromuscular junction (NMJ) is an early event in amyotrophic lateral sclerosis (ALS), ultimately leading to motor dysfunction and lethal respiratory paralysis. The hexanucleotide GGGGCC repeat expansion in the C9orf72 gene is the most common genetic mutation, and the dipeptid...

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Veröffentlicht in:	Molecular therapy 2024-07, Vol.32 (7), p.2176-2189
Hauptverfasser:	Sun, Shuangshuang, Shen, Yihui, Zhang, Xu, Ding, Ning, Xu, Zhe, Zhang, Qijie, Li, Lei
Format:	Artikel
Sprache:	eng
Schlagworte:	Agrin - genetics Agrin - metabolism amyotrophic lateral sclerosis Amyotrophic Lateral Sclerosis - drug therapy Amyotrophic Lateral Sclerosis - genetics Amyotrophic Lateral Sclerosis - metabolism Animals Antibodies - pharmacology C9orf72 C9orf72 Protein - genetics C9orf72 Protein - metabolism dipeptide repeat proteins Disease Models, Animal Humans LDL-Receptor Related Proteins - genetics LDL-Receptor Related Proteins - metabolism Longevity - drug effects Mice Mice, Transgenic Motor Neurons - drug effects Motor Neurons - metabolism MuSK neuromuscular junction Neuromuscular Junction - drug effects Neuromuscular Junction - metabolism Receptor Protein-Tyrosine Kinases - genetics Receptor Protein-Tyrosine Kinases - metabolism Receptors, Cholinergic - genetics Receptors, Cholinergic - metabolism
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