HUNTINGTON’S DISEASE

Description
The Huntington’s disease (HD) program is focused on developing therapeutic short interfering RNA (siRNA) targeting the gene that encodes the HD protein. The siRNA will be expressed from an AAV-vector. RNA interference (RNAi) is a mechanism used by cells to regulate the expression of genes and replication of viruses. The RNA interference mechanism uses siRNA to induce the destruction of target RNA using naturally occurring cellular protein machinery.