Article

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

2019-10-25

Abstract excerpt

<h4>Background</h4> 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. <h4>Methods</h4> We generated non-integrated...

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Literature Corpus work: e15d3202-40c0-5433-867e-7bc41e8fb485
DOI: 10.1101/801415

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