Improving induction efficiency, safety and cost by reprogramming mammalian fibroblasts to induced multipotent neural stem cells using small-molecule compounds.
BACKGROUND Induced neural stem cells provide opportunities for research applications in: • Drug screening and development • Diagnostic product development • Regenerative cell therapy products Despite remarkable progress in stem cell research, clinical use of induced stem cells poses ethical issues (eg use of embryonic stem cells) and potential safety risks (eg use of oncogenic transcription factors). In addition there is low reprogramming efficiency of stem cells. TECHNOLOGY Human and mouse fibroblasts and human urine cells can be fully reprogrammed into induced multipotent neural stem cells using small-molecule compounds. Stage of development: preclinical (in vitro).
• Unlimited cell resource • No genetic footprint: (ie chemical reprogramming) without the use of transcription factors • Small molecules are non-immunogenic • Efficient: ~2% fibroblasts form multipotent neurospheres • Effective: 95% fibroblasts reprogram to neural stem cells • Reproducible: Independently verified technique • Cost effective: A cell culture method using small-molecule compounds • Fast: Method (7-14 days)
Potential applications and markets include: • Research reagents • Diagnostics (eg drug screening) • Disease modelling • Pharmaceuticals • Neural stem cell-based treatment for neurodegenerative diseases.