Gene Therapy for Parkinsons and Alzheimers Diseases: from the Bench to Clinical Trials

Rui      Jorge Nobre; Luis      Pereira de Almeida

Abstract

Alzheimers and Parkinsons diseases represent the most prevalent neurodegenerative disorders worldwide. Current pharmacological or surgical treatments provide symptomatic benefits, particularly in the early stages, but none can delay or stop the progression of these diseases. There is an urgent need for new therapies able to modify disease progression. Gene therapy, mainly based on viral vectors, is presently being assumed as an important alternative to conventional treatments. After decades of preclinical developments, we are now facing an important period, in which several viral vector – mediated gene therapies are being tested in Phase 1 and Phase 2 clinical trials, with some of them showing promising results. This review intends to present an overview of the current efforts in the field for the treatment of Alzheimers and Parkinsons diseases.

Keywords: Parkinson's disease, Alzheimer's disease, neurodegenerative, gene transfer, molecular therapy, viral vectors, AAV, lentivirus, neurotrophic factors, genetic immunization