Teaching An Old Protein New Tricks: Developing A Novel Gene Therapy For Huntington's Disease
Friday, October 12, 2012 - 21:01
in Health & Medicine
Huntington’s disease is a neurodegenerative disorder caused by a genetic defect in the Huntington (HTT) gene. Huntington’s disease belongs to an elite class of diseases which are ‘monogenic’- directly attributable to one gene- and ‘dominant’, meaning that the presence of even one mutant HTT gene can have catastrophic consequences for those carrying the defective gene. A child born to a parent with Huntington’s disease has a 50% chance of getting the disease, depending on which copy of the Huntington gene is inherited from the affected parents. The disease is most prevalent among Europeans and North Americans, but still relatively rare, affecting approximately 40-100 individuals per million. read more