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Article

Targeted gene correction and functional recovery in achondroplasia patient-derived iPSCs.

Stem cell research & therapy - 28 Aug 2021

Zou Huan, Guan Mingfeng, Li Yundong, Luo Fang, Wang Wenyuan, Qin Yiren

Abstract excerpt

BACKGROUND: Achondroplasia (ACH) is the most common genetic form of dwarfism and belongs to dominant monogenic disorder caused by a gain-of-function point mutation in the transmembrane region of FGFR3. There are no effective treatments for ACH. Stem cells and gene-editing technology provide us with effective methods and ideas for ACH research and treatment. METHODS: We generated non-integrated iPSCs from an ACH...

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Identifiers

PMID: 34454631
DOI: 10.1186/s13287-021-02555-8
PMCID: PMC8403427

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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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