Developing Gene Silencing for the Study and Treatment of Dystonia
Abstract
Dystonia is a disabling and incurable neurological disorder characterized by twisting movements that cause significant disability. Some forms of dystonia are caused by genetic mutations and, therefore, passed on from generation to generation. The most common form of early onset genetic dystonia is a disease known as DYT1, in which children around age ten develop dystonia, usually in a leg or arm, and over 2 or 3 years spreads to affect all body parts causing substantial disability. There is no cure for DYT1. Therefore, the development of new treatments for DYT1 is a priority in dystonia research.
Document Details
- Document Type
- Technical Report
- Publication Date
- Oct 01, 2016
- Accession Number
- AD1017037
Entities
People
- Pedro Gonzalez-alegre
Organizations
- Children's Hospital of Philadelphia