Technology ID

A Neural Stem Line from a Niemann Pick C (NPC) Type 1 Patient for Therapy Development

Lead Inventor
Malik, Nasir (National Inst of Arthritis and Musculoskeletal and Skin Diseases)
Research Materials
Therapeutic Areas
Metabolic Disease
Development Stages
Pre-Clinical (in vitro)
Research Products
Human Cell Lines
Lead IC

This technology includes a neural stem cell (NSC) line derived from a Niemann Pick C (NPC) patient, aimed at advancing research and drug development for NPC, an inherited neurodegenerative disorder characterized by the accumulation of cholesterol in neurons. The NSCs, which serve as a crucial intermediate cell type, can be differentiated into any neuronal or glial cell of the brain or spinal cord under appropriate culture conditions. These cells originate from fibroblasts reprogrammed into induced pluripotent stem cells. Their ability to differentiate into various central nervous system cell types is vital for understanding NPC's underlying causes. Additionally, these NSCs are uniquely positioned for high-throughput drug screening, with the potential to identify novel compounds for treating NPC. This technology is particularly groundbreaking as it represents the first reported NSC line from an NPC patient, offering significant opportunities for novel discoveries in the disease's study.

Commercial Applications
The differentiated neural stem cells can be used to study the mechanisms underlying Niemann Pick C disease and employing these cells in high-throughput drug screening to identify novel treatment options for this neurodegenerative disorder.

Competitive Advantages
This neural stem cell technology represents a unique and groundbreaking advancement, offering unprecedented opportunities for in-depth study of the disease and the potential discovery of novel treatments through high-throughput drug screening.
Licensing Contact:
Knezevic, Vladimir