Abstract
The search for effective therapy of neurodegenerative diseases is an important issue and an important challenge. Nowadays, there are high hopes for the gene therapy based treatment. This involves using a noninfectious virus administered directly into the brain. AAV vectors are increasingly being used as tools for delivery of therapeutic agents to the CNS. The present paper aims to review clinical trials carried out so far with the use of the AAV vectors in treatment of neurodegenerative disorders: Parkinson’s disease, Alzheimer’s disease, amyotrophic lateral sclerosis, Canavan disease and late infantile neuronal ceroid lipofuscinosis. Results obtained from these trials show good tolerance of AAV vectors in the CNS and safety of therapies.