Abstract

Gene Therapy For Neurological Diseases

Shin-ichi Muramatsu , Professor of Neurology, Jichi Medical University (JMU)

Adeno-associated virus (AAV) vectors have been widely used for gene therapy. In Clinical studies on advanced Parkinson’s disease, investigators, including us, have shown the motor symptoms to be ameliorated along with an increased dopaminergic activity on positron emission tomography after gene transfer of aromatic L-amino acid decarboxylase (AADC) into the putamen. The beneficial effects of AADC gene transfer have also been shown in children with AADC deficiency. AAV vectors can achieve global transduction of the central nervous system by intravenous or intrathecal injection. We have been developing gene therapy for various diseases including sporadic amyotrophic lateral sclerosis, spinocerebellar ataxia 1, glucose transporter 1 deficiency, and GM2 gangliosidosis.