Conversion of human stem cells to dopaminergic neurons using a single, high-efficiency factor
JHU REF: [C12596]
Invention novelty: To convert human stem cells into dopaminergic neurons at high efficiency.
Value Proposition
As many as one million Americans suffer from Parkinson's disease (Parkinson’s Disease Foundation, 2012). Cell replacement therapy is a potential strategy to counter progressive neurological diseases, such as Parkinson’s disease, resulting from degeneration and loss of dopaminergic neurons. Recent advances, however, provide new possibilities to patients experiencing relatively selective neuronal cell loss. The opportunity becomes available through generation of dopaminergic (DA) neurons, which can be used for autologous transplantation. Previous attempts to yield DA neurons have resulted in inconsistent, incomplete conversion rates, leading to considerable safety concerns.
Advantages of this technology include:
- requires only one major single factor for stem-cell-to-neuron conversion
- can generate dopaminergic neurons with >80% purity
Technical Details
This invention is a method of converting human stem cells to dopaminergic neurons. Specifically, a single defined factor is used to induce highly efficient conversion of human stem cells to lineage-specific dopaminergic neurons. The established strategy is novel and highly applicable for disease modeling and cell replacement therapy for several neurological disorders, including but not limited to Parkinson’s disease, spinal cord injury, amyotrophic lateral sclerosis and hearing loss.
Looking for Partners: To develop & commercialize the technology as methods for neuron generation
Stage of Development: Pre-Clinical
Data Availability: Under CDA / NDA
Patent Status: Granted U.S. Patent 11,104,881
Publication(s)/Associated Cases: PMID: 49204172
Categories: Physical sciences
Keywords: neuron, stem cell
