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The Parkinson's Institute Awarded $1.3 Million Grant For Novel Approach For Gene Repair
Published Tuesday, February 1, 2011 4:00 am

The California Institute for Regenerative Medicine (CIRM) awarded The Parkinson's Institute and Sangamo BioSciences Inc. (Nasdaq: SGMO), $1.3 million for what experts consider “one of the next frontiers in the study of genetic diseases.” 

This grant funds research to correct genetic mutations that can lead to Parkinson’s disease. To accomplish this, scientists at The Parkinson’s Institute will use proteins called zinc-finger nucleases (ZFNs), developed by Sangamo BioSciences.  ZFNs can act as molecular “scissors,” attaching to a single location among the three billion segments in the human genome and then inducing the cell to repair this “cut” with a “corrected” segment. These newly corrected cell lines will provide new cellular tools to understand the mechanism for all forms of Parkinson’s disease and potentially other genetic mutation disorders. The ZFN technology will be applied to stem cells that were directly derived at The Parkinson’s Institute from skin cells of patients with Parkinson’s disease.

The grant was awarded the highest score of the 226 grant applications received by CIRM, from California’s top research institutions. Reviewers of the proposal unanimously agreed that this application addresses a significant translational bottleneck and will have a major impact on mutation repair.  CIRM reviewers were enthusiastic about this proposal to generate novel cellular tools to study the molecular basis of Parkinson’s disease and screen drugs and were impressed by the overall approach and are quoted “this project has the potential to bring about paradigm-shifting discoveries.”

Dr. J. William Langston, CEO of The Parkinson's Institute and the project’s principal investigator, says “It is a true honor to achieve the highest score in the state.  This research advances our search for a cure for the 1.5 million American’s suffering with Parkinson’s disease.”

The Parkinson’s Institute and Sangamo BioSciences are very excited to continue this partnership.  Working with Sangamo BioSciences will allow for optimization of the ZFN technique, specific for Parkinson’s research.  This collaboration highlights the strength of biotech research in California.

About the Parkinson’s Institute and Clinical Center

Founded in 1988, The Parkinson’s Institute and Clinical Center is America’s only independent non-profit organization that provides both basic and clinical research, clinical trials and a comprehensive movement disorder patient clinic for Parkinson’s disease and related neurological movement disorders, all under one roof.  Its mission is to discover the cause(s) of Parkinson’s disease, develop treatments that slow, halt, or reverse the progression of the disease, as well as to provide the highest quality compassionate care for individuals with Parkinson’s disease and related movement disorders.  Its unique freestanding organization supports a strong collaboration of translational medicine designed to more directly connect research to patient care – from the "bench to bedside." For more information, please call 408.734.2800

About Sangamo BioSciences

Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and modification.  The most advanced ZFP Therapeutic® development program is currently in a Phase 2b clinical trial for evaluation of safety and clinical effect in patients with diabetic neuropathy and a Phase 2 trial in ALS. Sangamo also has a Phase 1 / 2 clinical trial and two ongoing Phase 1 clinical trials to evaluate safety and clinical effect of a treatment for HIV/AIDS as well as a Phase 1 trial of a treatment for recurrent glioblastoma multiforme. Other therapeutic programs are focused on Parkinson’s disease, monogenic diseases, neuropathic pain and nerve regeneration.  Sangamo’s core competencies enable the engineering of a class of DNA-binding proteins known as zinc finger DNA-binding proteins (ZFPs).  By engineering ZFPs that recognize a specific DNA sequence Sangamo has created ZFP transcription factors (ZFP TFs) that can control gene expression and, consequently, cell function.  Sangamo is also developing sequence-specific ZFP Nucleases (ZFNs) for gene modification.  Sangamo has established strategic partnerships with companies in non-therapeutic applications of its technology including Dow AgroSciences and Sigma-Aldrich Corporation. For more information about Sangamo, visit the company’s website at www.sangamo.com
ZFP Therapeutic® is a registered trademark of Sangamo BioSciences, Inc.

About California Institute for Regenerative Medicine

CIRM was created by the people of California in 2004 by passage of Proposition 71, to provide funding to advance stem cell research as a source of medical advancement and scientific knowledge to relieve human suffering from chronic disease and injury, and to create jobs within California in advancing these goals. www.cirm.ca.gov

Media Contact:
Mary Tunison
Development Director
The Parkinson's Institute and Clinical Center

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