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Article

Cell type-specific gene therapy confers protection against motor neuron disease caused by a TFG variant.

Proceedings of the National Academy of Sciences of the United States of America - 19 Nov 2024

Lettman Molly M, Mendina Caitlin A, Burkard Emma, Alvin James R, Zhu Yunyun, Coon Joshua J, Audhya Anjon

Abstract excerpt

Inherited forms of motor neuron disease (MND), including hereditary spastic paraplegias (HSP), are associated with the death or dysfunction of nerve cells that control skeletal muscle activity. However, in some cases, the impacts of genetic variants underlying MND act in a non-cell autonomous manner, instead affecting the function of other cell types necessary for neuronal maintenance. Pathological mutations in...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 39527745
DOI: 10.1073/pnas.2410996121
PMCID: PMC11588061

Genes

TFG
GFAP

Variants

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

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