Gene-Edited IPSCs

Gene-edited iPSCs (induced pluripotent stem cells) are iPSCs that have been modified using precise gene-editing technologies such as CRISPR/Cas9, TALENs, or zinc-finger nucleases (ZFNs). These cells combine the advantages of iPSCs—pluripotency and differentiation potential—with targeted genetic modifications.

Content 

• Reprogramming:
  • Derive iPSCs from patient somatic cells or commercially available sources.
• Gene Editing:
  • Design: Target selection using bioinformatics tools to minimize off-target effects.
  • Editing Tools:
    • CRISPR/Cas9 plasmid or ribonucleoprotein (RNP) delivery.
• • Delivery Methods:
    • Electroporation, nucleofection, or viral vectors.
  • Selection:
    • Antibiotic resistance or fluorescence-based sorting to isolate edited cells.

Applications

• Disease Modeling:
  • Reproduce disease-relevant mutations in iPSCs to study pathogenesis.
  • Generate isogenic controls for comparative studies.
• Therapeutic Development:
  • Test gene-corrected iPSCs for cell replacement therapies.
  • Screen for drug candidates using gene-edited iPSC-derived cell models.
• Functional Genomics:
  • Study gene function and regulatory networks through knockouts, knock-ins, or base editing.
• Precision Medicine:
  • Model patient-specific genetic variants for personalized treatment strategies.

Gene-edited iPSCs represent a groundbreaking convergence of stem cell and gene-editing technologies. Their ability to mimic genetic diseases, explore functional genomics, and facilitate therapeutic development positions them as pivotal tools in advancing precision medicine and biotechnology research.