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Nurr1: A Novel Target for the Treatment of Catecholamine Related Diseases

Nurr1 is a polypeptide that upregulates tyrosine hydroxylase activity and promotes expression of DOPA, epinephrine and norepinephrine. This could be useful for the treatment of catecholamine related diseases, such as Parkinson's, manic depression, and schizophrenia.   

INVENTION: The invention is based on the discovery that expression of Nurr1 polypeptide induces tyrosine hydroxylase in both undifferentiated and differentiated mammalian cells including adult hippocampal progenitor cells. Increased tyrosine hydroxylase activity promotes the expression of DOPA, norepinephrine and epinephrine. Cells expressing Nurr 1 can be used to treat catecholamine deficiency diseases directly. In addition, these diseases can be treated by transfection using the genetic construct for Nurr1 expression.  


  • Experimental platform to model tyrosine hydroxylase-related human disease
  • Identification of tyrosine hydroxylase-related deficiencies
  • Novel treatment for catecholamine-related deficiencies, such as Parkinson's disease, manic depression and schizophrenia


  • The discovery provides new target for development of novel treatments for catecholamine-related diseases

STAGE OF DEVELOPMENT:   Preclinical. In vitro data. 

BACKGROUND: During development, neural stem cells differentiate into the different types of neurons and glia found in the adult central nervous system (CNS) and peripheral nervous system (PNS). These different types of neurons are classified based on the particular types of neurotransmitters they produce. For example, dopaminergic neurons produce dopamine, while noradrenergic neurons produce norepinephrine. The neurotransmitters dopamine and norepinephrine belong to a class of compounds called catecholamines. Regulation of tyrosine hydroxylase expression and catecholamine related diseases, including Parkinson's disease, manic depression and schizophrenia, are poorly understood.

INVENTORS: Fred H. Gage, Kazuhiro Sakurada and Theo Palmer
PATENT STATUS: U.S. Patent No. 6,312,949 issued November 6, 2001
PUBLICATION: Development 126(18):4017 (Sept. 1999)
CONTACT: Kiren Rockenstein, PhD, 858 453 4100 x1758;
Patent Information:
For Information, Contact:
Kiren Rockenstein
Licensing Associate
Salk Institute
Gene Therapy
Manic depression
Neurodegenerative Disorders
Regenerative Medicine
Stem Cells
Tyrosine hydroxylase
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