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Article

Therapeutic strategy for spinal muscular atrophy by combining gene supplementation and genome editing.

Nature communications - 24 Jul 2024

Hatanaka Fumiyuki, Suzuki Keiichiro, Shojima Kensaku, Yu Jingting, Takahashi Yuta, Sakamoto Akihisa, Prieto Javier, Shokhirev Maxim, Nuñez Delicado Estrella, Rodriguez Esteban Concepcion, Izpisua Belmonte Juan Carlos

Abstract excerpt

Defect in the SMN1 gene causes spinal muscular atrophy (SMA), which shows loss of motor neurons, muscle weakness and atrophy. While current treatment strategies, including small molecules or viral vectors, have shown promise in improving motor function and survival, achieving a definitive and long-term correction of SMA's endogenous mutations and phenotypes remains highly challenging. We have previously developed...

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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: 39048567
DOI: 10.1038/s41467-024-50095-5
PMCID: PMC11269569

Genes

SMN1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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