UMIP-82 – Activation or Suppression of Cryptic Amyloidogenic Elements (CAE) in Untranslated Genomic Areas for Therapeutic Purposes

Problem: Whereas in some conditions such as Alzheimer’s disease amyloid formation is deleterious, amyloids have been found to put cancerous tumors into a state of dormancy or kill them altogether. Genes containing “cryptic amyloidogenic elements” or “CAE” are highly expressed in some cancers including lung, prostate, breast, liver, esophageal, colorectal, pancreatic, bladder, kidney, ovary, stomach, brain and skin – a list that includes six of the ten least successfully treated forms. There is a particular need for treatments for difficult to treat cancers. Metastasis prevention in all cancers is also a critical need. There is also a need to deliver cancer treatment beyond the blood brain barrier, which gene therapy can do. Technology: Drs. Zuchner and Rebelo have determined that previously unrecognized CAE are expressed under certain circumstances (e.g., familial mutation), leading to aggregation and neuronal degeneration in certain familial neurodegenerative diseases. Available for licensing is an innovative technology that comprises the use of these cryptic amyloidogenic elements, including nucleic acids that encode them, to inhibit the growth and survival of cancerous cells. The methods include an in vivo gene editing technique / gene therapy that activates the CAE and can cross the blood-brain barrier to treat gliomas. Peter Gutenberg pxg372@miami.edu 305-243-4604