Small molecule regulators of autophagy identified by an image-based high throughput screen

Summary Compounds for the induction of autophagy and screening methods for further identification of autophagy inducing compounds.

Autophagy is a lysosome-dependent process whereby proteins or damaged organelles within a cell are degraded. During this process, an autophagosome encloses the component of the cell to be degraded, and the vesicle fuses with a lysosome, which degrades the component. Autophagy is primarily involved in the degradation of long-lived proteins, and appears to have great significance for the treatment of diseases associated with improper protein folding, such as Huntington’s disease.

Applications Autophagy mediates the degradation of long-lived proteins and misfolded proteins within the cell. A high concentration of misfolded proteins is a prominent feature of many neurodegenerative diseases. For example, the pathogenic mechanism of Huntington’s disease is that a large amount of polyglutamine (polyQ) protein accumulates in the neurons and cannot be cleared away. In this situation, autophagy is regarded as a mechanism for clearing away polyQ.

Compounds that stimulate autophagy in neurons and other cells may be used to treat a host of neurodegenerative diseases, such as Alzheimer’s, Parkinson’s ALS, and Huntington’s disease. 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



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