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MCL 3797.0: Neural Stem Cells Derived from Induced Pluripotent Stem Cells

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

Background and Description

In recent years, techniques have been developed to reprogram differentiated adult cells into pluripotent stem cells. The resulting cells, known as induced pluripotent (iPS) cells, are widely viewed as being potentially valuable both as research tools and for possible therapeutic purposes because they are expected to be more widely available than human embryonic stem cells, with fewer of the ethical issues affecting embryonic cells. McLean investigators, working with the Jaenisch laboratory at the Whitehead Institute of MIT, have demonstrated that iPS cells can be differentiated into neural stem cells, which can be used to treat symptoms of Parkinson's Disease (PD) in animal models. The investigators created iPS cells from fibroblasts and then induced them to differentiate into neural precursor cells. When grafted into the brains of rats experimentally induced to exhibit PD-like symptoms the transplanted cells were found to have extended into the surrounding brain tissue and were able to improve the PD-like symptoms.

Potential Commercial Uses

These "proof-of-concept" animal studies lay the groundwork for the use of stem cell therapies, particularly those relying on iPS cells, for the treatment of humans afflicted with disorders such as PD. The invention enables adult cells harvested from skin or other non-neuronal tissue, to be easily and reliably reprogrammed to become 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 a patient's own cells, or eventually a standardized product for heterologous transplants. This invention will lead to safer, more specific, and more powerful treatments for PD, which would be of great importance given the prevalence of this disease in our aging populations. Current therapy for PD, such as the pharmacological use of the dopamine precursor L-DOPA to replace brain dopamine, has had only limited effectiveness and will produce side-effects for patients

Publication and Patent Status

A U.S. patent application claiming this invention has been filed. The research has been published as Wernig, et al., 2008 PNAS 105(15):5856-61.

Licenses Available

McLean Hospital and the Whitehead Institute are 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