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Article

Generation of two induced pluripotent stem cell lines from spinal muscular atrophy type 1 patients carrying no functional copies of SMN1 gene.

Stem cell research - 1 Jun 2023

Zeng Wenshu, Kong Xiaohui, Alamana Christina, Liu Yu, Guzman Jessica, Pang Paul D, Day John W, Wu Joseph C

Abstract excerpt

Spinal muscular atrophy (SMA) is a severe neurodegenerative muscular disease caused by the homozygous loss of survival of motor neuron 1 (SMN1) genes. SMA patients exhibit marked skeletal muscle (SKM) loss, eventually leading to death. Here we generated two iPSC lines from two SMA type I patients with homozygous SMN1 mutations and validated the pluripotency and the ability to differentiate into three germ layers....

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Source

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Identifiers

PMID: 37087898
DOI: 10.1016/j.scr.2023.103095
PMCID: PMC11068589

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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