In vivo Gene Therapy for Neuronopathic Disorders

Currently, no therapeutics alter the disease progression of major neurodegenerative disorders of adults or of childhood.  A major hurdle in the development of such therapeutics is the difficulty in delivering drugs to the brain through the blood brain barrier.  Recent advances in gene therapy technology now afford platforms that enable broad delivery across regions of the CNS.  I will describe the challenges that remain, the uses of AAV therapeutics, and existing clinical approaches that are ongoing. 
Install Google Tag Manager Copy the code below and paste it on to every page of your website. Paste this code as high in the of the page as possible: Additionally, paste this code immediately after the opening tag: For more information about installing the Google Tag Manager snippet, visit our Quick Start Guide .