SUMMARY OF INVENTION
Patient-specific induced pluripotent stem cells (iPSCs) are a powerful tool for modeling disease phenotypes in vitro, and they have been successfully used to study early-onset disorders. Their value in modeling late-onset neurodegenerative diseases such as Parkinson’s or Alzheimer’s, in contrast, has been limited to date.
One problem has been the embryonic nature of iPSc-derived midbrain dopamine neurons. This prevents them from fully recapitulating the severe degenerative pathology of these diseases.
This invention facilitates more accurate modeling of late-onset neurodegenerative disorders through a novel method that manipulates the cellular “age” of iPSc-derived dopamine neurons in vitro. The technology starts by reprogramming cell cultures into a de-differentiated state, and then inducing “aging” through the reintroduction of age-related markers that can control and accelerate the timeline.
- As a research tool, this method permits enhanced ability to model late-onset neurodegenerative diseases
- Potential to produce a scalable supply of “aged” iPSCs for use in high throughput screening and other applications
- Developed by outstanding scientific team with proven experience in stem cell innovation
In the U.S. alone, there are an estimated 5.3 million patients with Alzheimer’s and another 1 million with Parkinson’s disease; the prevalence of these conditions will only increase as the population ages. With no adequate treatments to restore proper dopaminergic neuron function, this is a high-priority research field which could benefit from a method that readily and accurately produces “aged” iPSCs.
AREAS OF APPLICATION
Research tool for use in modeling neurodegenerative diseases
STAGE OF DEVELOPMENT
Proof-of-concept in vitro
Applications pending in U.S., Europe, Canada, Australia, and Japan
Lorenz Studer, MD/PhD, Director, Center for Stem Cell Biology, Memorial Sloan Kettering Cancer Center