Fig. 6
From: Targeted gene correction and functional recovery in achondroplasia patient-derived iPSCs
Diagrammatic strategy of ACH stem cell research. Somatic cells can be isolated and cultured from ACH patient-derived adipose, skin, and urine, and can further be reprogrammed into iPSCs. After gene correction of iPSCs or AD-MSCs via CRISPR-Cas9, they can be differentiated into healthy cells, such as MSCs or chondrocyte precursor cells. Then these healthy cells can be transplanted into ACH mouse models to assess their relative safety and therapeutic effects. The clinical application of ACH patient-derived stem cells will be pursued in the future