Yihong Hu, PhD
Research Fellow, Drug Discovery Group
McColl-Lockwood Laboratory for Muscular Dystrophy Research
Department of Neurology
PhD: 2006, Institute of Biochemistry and Cell Biology, Chinese Academy of Sciences (Shanghai, China)
MS: 2001, Henan Normal University (Xinxiang, China)
BS: 1998, Henan Normal University, (Xinxiang, China)
Drug Discovery for Muscular Dystrophies
Dr. Hu is interested in discovering drugs to treat muscular dystrophies including Fukuyama congenital muscular dystrophy (FCMD), limb-girdle muscular dystrophy type 2I (LGMD2I) and Duchenne muscular dystrophy (DMD). Currently, there are no effective drugs and treatment available for any form of muscular dystrophies.
Overexpression of a glycosyltransferase gene, called LARGE, in muscles, can functionally restore the glycosylation of α-dystroglycan (α-DG). It is hypothesized that the up-regulation of glycosylation pathways by small compounds or natural products will rescue hypoglycosylation-related muscular dystrophies by enhancing glycosylation of α-DG. Dr. Hu has been actively involved in developing cell-based assays for high throughput screening of small compound libraries. Several lead compounds have been identified. Currently, those compounds are under validation in dystrophic mdx mouse model.
DMD is an X-linked fatal muscle wasting disease caused by mutations in the dystrophin gene. Recent developments have shown that antisense oligonucleotide-mediated exon skipping can effectively restore the expression of dystrophin protein and muscle functions. It is possible that exon skipping can also be achieved by small compounds. Using the exon skipping screening systems established in the McColl-Lockwood Laboratory, drug screening is being undertaken to identify small compounds capable of inducing exon skipping. Lead compounds have been identified. These candidate compounds will be validated using in vitro cell culture system specifically designed to test the skipping of human DYSTROPHIN exon 50 and mouse DYSTROPHIN exon 23.
Hu Y, Ying H, Xu Y. hF-LANa, a human homologue of Derlin family, regulating the expression of cancer-related genes promotes NIH3T3 cell transformation. Cancer Lett 2007; 258: 171-180. [PMID: 17977649]
Hu Y, Fang C, Xu Y. The effect of isoforms of the cell polarity protein, human ASIP, on the cell cycle and Fas/FasL-mediated apoptosis in human hepatoma cells. Cell Mol Life Sci 2005; 62: 1974-1983. [PMID: 16091846]