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Intrathecal (G <sub>4</sub> C <sub>2</sub> ) <sub>149</sub> delivery in C9orf72-deficient mice yields mild motor dysfunction and ALS/FTD pathological hallmarks

2026-02-11

Abstract excerpt

<h4>SUMMARY</h4> A repeat expansion in C9orf72 is the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), yet existing mouse models incompletely engage spinal regions implicated in disease. Here, an adeno-associated virus encoding (G 4 C 2 ) 149 repeats was delivered via neonatal intrathecal injection, achieving widespread CNS expression with robust spinal cord...

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Literature Corpus work
b18a411b-87ca-51d9-ad59-2572cb0de5f6
DOI
10.64898/2026.02.09.704060
Open publication

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Intrathecal (G <sub>4</sub> C <sub>2</sub> ) <sub>149</sub> delivery in C9orf72-deficient mice yields mild motor dysfunction and ALS/FTD pathological hallmarksDOI 10.64898/2026.02.09.704060
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