Featuring of Prof. Edwin CHAN’s work on the treatment of rare neurodegenerative diseases at the Hong Kong Research Grants Council YouTube Channel
Keywords: Huntington's disease, Polyglutamine disease, Spinocerebellar ataxia, peptide, small molecule
Professor Edwin Chan from the School Life Sciences from The Chinese University of Hong Kong is working on rare neurodegenerative diseases including the Huntington’s Disease (HD). He recently developed a new inhibitor for the treatment of HD. This patented technology is known as P3. P3 is a 13-amino acid peptidylic inhibitor that can potently suppress expanded CAG (Glutamine) RNA-induced nucleolar stress, and rescue neurodegeneration in animal disease model and cell death in patient fibroblasts. The above work has recently been featured at the Hong Kong Research Grants Council YouTube Channel.
The core benefits of this technology are:
(1) the inhibitor is capable of directly interacting with and neutralizing the toxic expanded CAG RNA;
(2) the mechanism of this inhibitor is well-defined that it reduces cellular p53 level and suppresses caspase activities; and
(3) this invention has been proven to have the potential to treat other polyglutamine diseases, such as spinocerebellar ataxias (SCA).
Related patent and references:
(1) US9,297,798 (http://www.google.com/patents/US9297798)
(2) US Provisional patent (Priority date: 23 Dec 2015)
(3) US non-Provisional patent (Priority date: 17 Feb 2016)
(4) Zhang et al, Dis. Model. Mech, 2016 (http://www.ncbi.nlm.nih.gov/pubmed/26839389)
(5) Tsoi et al, PNAS, 2012 (http://www.ncbi.nlm.nih.gov/pubmed/22847428)
Introduction brochure (download):