Short G-rich oligonucleotides as a potential therapeutic for Huntington's Disease |
| |
Authors: | Michael Skogen Jennifer Roth Sarah Yerkes Hetal Parekh-Olmedo Eric Kmiec |
| |
Institution: | (1) Department of Biological Sciences, University of Delaware, Delaware Biotechnology Institute, 15 Innovation Way, Newark, DE 19711, USA |
| |
Abstract: | Background Huntington's Disease (HD) is an inherited autosomal dominant genetic disorder in which neuronal tissue degenerates. The pathogenesis
of the disease appears to center on the development of protein aggregates that arise initially from the misfolding of the
mutant HD protein. Mutant huntingtin (Htt) is produced by HD genes that contain an increased number of glutamine codons within
the first exon and this expansion leads to the production of a protein that misfolds. Recent studies suggest that mutant Htt
can nucleate protein aggregation and interfere with a multitude of normal cellular functions. |
| |
Keywords: | |
本文献已被 SpringerLink 等数据库收录! |