Small Molecule Therapeutic and Screening Assays for Drugs Active Against Chronic Neural Degenerative Diseases.

Summary Huntington's disease, as well as other ataxias, are caused by the abnormal expansion of nucleic acid CAG repeats that code a glutamine stretch. These repeats are found within several otherwise non related proteins. The abnormally long polyglutamine tracts are believed to trigger programmed cell death (apoptosis), neuronal death, and subsequent neural degenerative disease.

As part of an ongoing research effort at Harvard University, Harvard Medical School researchers have been investigating the biology of caspases, key components of cellular apoptosis systems. It has been found that caspases are directly involved in the cleavage of aberrant proteins such as those containing expanded polyglutamine tracts, and it is believed that the caspase mediated cleavage of these aberrant proteins leads to premature neuron death. Therefore, drugs that block the caspase mediated cleavage may enhance neuronal survival. Experimentally, Harvard researchers have found this to be the case.

Applications - a method of screening for inhibitors of caspase mediated neuronal cell death. - a small molecule therapeutic that was shown to enhance neuronal survival for cells containing expanded polyglutamine repeats. These are found in neurodegenerative diseases such as Huntington's. For Further Information Please Contact the Director of Business Development Michal Preminger Email: [email protected] Telephone: (617) 432-0920

Inventor(s): Yuan, Junying

Type of Offer: Licensing



« More Pharmaceutical Patents

---