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

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...

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Detalles Bibliográficos
Autores principales:	Zou, Huan, Guan, Mingfeng, Li, Yundong, Luo, Fang, Wang, Wenyuan, Qin, Yiren
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2021
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8403427/ https://www.ncbi.nlm.nih.gov/pubmed/34454631 http://dx.doi.org/10.1186/s13287-021-02555-8

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