CRISPR combined with stem cells

CRISPR combined with stem cells enables precise genome editing to model diseases, correct genetic defects, and enhance therapeutic potential in regenerative medicine. This synergy supports applications from basic research in differentiation pathways to clinical therapies like gene-corrected hematopoietic stem cells for blood disorders. https://pmc.ncbi.nlm.nih.gov/articles/PMC5651489/​

Key Applications

CRISPR/Cas9 facilitates gene knockouts, knock-ins, and activation in human embryonic stem cells (hESCs), induced pluripotent stem cells (iPSCs), and organoids, revealing essential genes for survival and lineage commitment. It creates isogenic disease models for drug screening, such as retinoblastoma or cystic fibrosis organoids.

https://pmc.ncbi.nlm.nih.gov/articles/PMC5651489/​

Therapeutic Advances

Gene correction in patient-derived iPSCs restores function in conditions like β-thalassemia or hemophilia, while CCR5 edits confer HIV resistance in stem cell-derived therapies. CAR-T cells from edited stem cells boost anti-tumor efficacy by disrupting PD-1.

https://pmc.ncbi.nlm.nih.gov/articles/PMC5651489/​

Research Tools

Dox-inducible CRISPRi/a systems control gene expression dynamically in neural and pluripotent stem cells, aiding studies of tumor tropism and mechanobiology. Genome-scale screens identify lncRNA loci and pharmacological profiles via reporter lines.

https://pmc.ncbi.nlm.nih.gov/articles/PMC5651489/​