4月16日研究生进展类讲座（医学）

        The focus of my research is the use of viral vector-mediated gene therapy to treat neurodegenerative diseases such as Amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA). ALS/MND is a common adult onset neurodegenerative disease characterised by progressive degeneration of motor neurones in the brainstem and spinal cord. There is no effective treatment or cure for MND/ALS and other neurodegenerative disorders (Spinal Muscular Atrophy, Parkinson disease and Huntington disease).

         Gene therapy approaches offer a promising strategy for delivery of genes to enhance motor neuron survival. New approaches for the treatment of neurological disorders have been developed by using lentiviruses and other viruses. These vectors have been refined to a very high safety and efficiency levels. Lentiviruses are particularly advantageous for use in gene transfer to the nervous system. Numerous animal studies have now been undertaken with these vectors and correction of disease models has been obtained.

        Recent studies have demonstrated very good efficacy of Lentiviral-mediated gene therapy approaches to ALS, SMA and other degenerative disorders. These studies provide great optimism for the future utility of lentiviral gene delivery as a therapeutic strategy for neurodegenerative diseases. One of my interests is focussing on PTEN/PI3 Kinase pathway and motor neuron survival in vitro and in vivo.