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Differentiation of Human Embryonic Stem Cells into Dopaminergic Nerve Cells

Posted Jun 22 2009 5:00pm

Description of Invention:
The invention described here is a novel method of differentiating human embryonic stem cells (hESCs) into dopaminergic nerve cells, which is preferable to the currently available dopaminergic differentiation techniques.

This invention potentially provides a source of sufficient dopaminergic cells not only for the clinical transplantation of dopaminergic tissue but also for in vitro studies of human cells useful for pharmaceutical screens related to neurodegenerative disorders and substance abuse.

Neurodegenerative disorders encompass a range of debilitating conditions including Parkinson's disease, Alzheimer's disease, and Huntington's disease. The primary cause of cognitive dysfunction for these three disorders has been directly linked to neuron degeneration, usually in specific areas of the brain. Transplantation of fetal dopaminergic neurons in affected areas of the brain in late stage Parkinson's disease has demonstrated clinical utility in human patients. However, fetal transplantation therapy generally requires human tissue from at least 3-5 embryos to obtain a clinically reliable improvement in the patient, thus demonstrating a need for a larger and more reliable source of dopaminergic cells. HESCs are a promising alternative source of cells because they can grow in culture indefinitely and have the ability to differentiate into a variety of cell types. One of the most efficient methods for conversion of hESCs to dopaminergic neurons requires the presence of mouse stromal cells which have an undefined dopaminergic inducing activity. However, the major disadvantage of this method is the exposure of hESC to mouse cells, which hinders any downstream clinical application due to possible transfer of animal cells and pathogens. This invention has unveiled the molecular nature of the activity of the mouse cells and established an efficient alternative approach for dopamine neuron generation, which is more suitable for clinical application. This innovative approach potentially provides a large and reliable source of dopaminergic cells sufficient for clinically relevant transplantation of dopaminergic tissue as well as in vitro pharmacologic studies of human dopaminergic cells.

  • Human dopaminergic cell source for neuronal transplantation, with potential clinical application to Parkinson's disease and possibly other neurodegenerative disorders.
  • Human dopaminergic cell source for in vitro models for pharmaceutical screens relevant to neurodegenerative disorders and substance abuse.

Development Status:
Early stage.

William Freed (NIDA)
Tandis Vazin (NIDA)

Patent Status:
HHS, Reference No. E-176-2008/0
PCT, Application No. PCT/US2009/065007 filed 18 Nov 2009

Relevant Publication:
  1. In preparation.

Licensing Status:
Available for licensing.

Collaborative Research Opportunity:
The National Institute on Drug Abuse, Development and Plasticity Section, is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize this technology. Please contact Vio Conley, M.S. at 301-496-0477 or for more information.

Central Nervous System
Central Nervous System - Therapeutics

For Additional Information Please Contact:
Norbert Pontzer Ph.D., J.D.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325 Room 23,
Rockville, MD 20852
United States
Phone: 301-435-5502
Fax: 301-402-0220

Ref No: 1976

Updated: 06/2009

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