Our first project is focused on developing a gene therapy for Huntington’s disease.
The project builds upon information found in the following publication, which is available as an open access journal article:
Grondin R, Kaytor MD, Ai Y, Nelson PT, Thakker DR, Heisel J, Weatherspoon MR, Blum JL, Burright EN, Zhang Z, and Kaemmerer, WF. Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum. Brain, 2012 Apr; 135 (Pt 4): 1197-1209. Epub 2012 Jan 16.
We have been funded by the Hereditary Disease Foundation for a project to investigate the efficacy of the same shRNA (that we found to be safe in the rhesus brain) in improving the “health” of cells derived from adult stem cells from persons affected with Huntington’s disease. The abstract describing the funded project, entitled “Efficacy of shRNA-mediated non-allele-specific partial suppression of huntingtin in patient-derived iPS cells” may be found on the web site of the Hereditary Disease Foundation, or by clicking here .
A pdf version of the poster we presented at the Hereditary Disease Foundation’s investigators’ conference, “HD2014: The Milton Wexler Celebration of Life” on August 6-9, 2014 in Cambridge, Massachusetts, may be found here.