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MCL 3661.0: Cell Sorting Codes for Purification of Neural Stem Cells and Stem Cell-Derived Neurons

Ole Isacson, M.D. Ph.D., et al.

Background and Description

Parkinson's Disease (PD) is a chronic, progressive motor system disorder affecting as many as one million Americans, and is one of several neurodegenerative diseases characterized by neuronal cell loss. Because the regenerative capacity of the adult brain is very limited, cell transplantation therapies have been used to treat neurodegenerative disease, with moderate success. However, widespread application of cell-based therapies will depend upon the availability of large amounts of neuronal precursor cells having consistent characteristics. Preferably, the precursor cell populations would be highly enriched for the therapeutic cell type, and cells that might cause deleterious effects such as neural tumors should be reduced or eliminated. Neural cells differentiated in vitro from human embryonic stem cells (hESCs) exhibit broad cellular heterogeneity which limits their usefulness. The invention is based on the discovery, isolation, and characterization of specific neural stem cells that are derived in vitro from hESCs, and also features a powerful methodology for sorting and purifying specific cell populations.

Potential Commercial Uses

The invention overcomes many of the hurdles seen to date in proposed cell therapy approaches, by providing homogeneous and well characterized cell populations, enriched in the cell types most likely to have therapeutic effects. Analysis of cell surface markers shows that these subpopulations contain a significant fraction of neurons or neuronal precursors, confirming their potential usefulness in cell therapy applications. The invention therefore enables preparation of homogeneous populations of neuronal precursor cells that can be used in cell therapy approaches for treatment or prevention of PD. Such methods can make use of autologous transplants using populations purified from a patient's own cells, or eventually a standardized product for heterologous transplants. The invention also features methods for identifying and sorting neuronal cell populations that are expected to have significant value in research and clinical applications.

Publication and Patent Status

A U.S. patent application claiming this invention has been filed. This research has been published as Pruszak, et al., Stem Cells;27(12):2928-40 (2009).

Licenses Available

McLean Hospital is offering a worldwide exclusive license to this technology.

For more information, please contact:

David J. Glass, Ph.D.
Senior Associate Director, Technology Transfer
McLean Hospital Research Administration
115 Mill Street
Belmont, MA 02478-9106
(617) 855-3825 - tel
(617) 855-3745 - fax