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Parkin and PINK1-Based Therapies for Parkinson's Disease and Other Mitochondrial Diseases

Posted Jun 13 2010 5:00pm

Description of Invention:
This technology provides methods for treating Parkinson's disease and other diseases associated with mitochondrial dysfunction.

Mutations in mitochondrial DNA (mtDNA) are responsible for a broad spectrum of inherited diseases, with symptoms that can range from mild to very severe. Accumulated mutations in mtDNA have also been linked to the pathogenesis of common diseases such as cancer, diabetes mellitus, and neurodegenerative disorders. In Parkinson's disease, for example, the accumulation of defective mitochondria appears to be responsible for the loss of midbrain neurons that produce dopamine neurotransmitter, which is a key feature of this disease.

In their recent work, Dr. Richard Youle and co-investigators have linked the fields of mitochondrial quality control and the genetics of Parkinson's disease. They have discovered that the Parkin protein is selectively recruited to damaged mitochondria, and promotes autophagic degradation of these mitochondria; ablation of Parkin increases levels of damaged mitochondria in cells. They have also discovered that another protein associated with mitochondrial disease, the mitochondrial PTEN-induced kinase-1 (PINK1), accumulates on the surface on damaged mitochondria, and that the presence of full-length PINK1 is necessary and sufficient for Parkin recruitment to the mitochondria. Thus, both Parkin and PINK1 play specific and important roles in mitochondrial quality control and disposal.

This technology describes methods of treating Parkinson's disease or other mitochondrial diseases such as KSS (Kearns Sayre syndrome), MERRF (Myoclonus epilepsy ragged-red fibers), MELAS (mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes), NARP (Neuropathy ataxia, retinitis pigmentosa), and LHON (Leber hereditary optic neuropathy) by increasing PINK1 or Parkin expression or activity, as well as methods of reducing the number of defective mitochondria in a cell by increasing PINK1 or Parkin expression or activity.

  • Development of therapies for Parkinson's disease and other diseases associated with mitochondrial dysfunction
  • Development of individualized treatment regimens for mitochondrial diseases through ex vivo or in vitro testing of candidate drugs

Richard J Youle (NINDS)

Patent Status:
HHS, Reference No. E-225-2009/0
US, Application No. 61/256,601 filed 30 Oct 2009

Relevant Publication:
  1. A Abeliovich. Parkinson's disease: Mitochondrial damage control. News and Views, Nature 2010 Feb 11:463:744-745. [ PubMed: 20148026 ]
  2. D Narendra et al. PINK1 is selectively stabilized on impaired mitochondria to activate Parkin. PLoS Biol. 2010 Jan 26;8(1):e1000298. [ PubMed: 20126261 ]
  3. D Narendra et al. Parkin-induced mitophagy in the pathogenesis of Parkinson disease. Autophagy. 2009 Jul;5(5):706-708. [ PubMed: 19377297 ]
  4. D Narendra et al. Parkin is recruited selectively to impaired mitochondria and promotes their autophagy. J Cell Biol. 2008 Dec 1;183(5):795-803. [ PubMed: 19029340 ]

Licensing Status:
Available for licensing.

Collaborative Research Opportunity:
The National Institute of Neurological Disorders and Stroke is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize methods of treating mitochondrial diseases by increasing PINK1 or Parkin expression or activity. Please contact Dr. Martha Lubet at 301-435-3120 or for more information.

Cancer - Therapeutics
Gene Based Therapies
Gene Based Therapies - Therapeutics
Internal Medicine
Internal Medicine - Therapeutics
Central Nervous System
Central Nervous System - Therapeutics
In-vitro Data

For Additional Information Please Contact:
Tara Kirby Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Phone: 301-435-4426
Fax: 301-402-0220

Ref No: 2121

Updated: 06/2010

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