Synergistic effects of treating the spinal cord and brain in CLN1 disease

• DOI: 10.1073/pnas.1701832114
• Publication Date: 2017-07-04T00:50:19Z
• Authors: Charles Shyng; Hemanth R. Nelvagal; Joshua T. Dearborn; Jaana Tyynelä; Robert E. Schmidt; Mark S. Sands; Jonathan D. Cooper
• Keywords: Batten disease; Neuronal ceroid lipofuscinosis

Significance Infantile neuronal ceroid lipofuscinosis (INCL, or infantile Batten disease) is a fatal childhood disease that devastates the brain. Trying to treat brain alone has so far proved ineffective, we looked for effects of in other parts body. Unexpectedly, found spinal cord severely affected before and contributes outcome. Directing gene therapy just INCL mice can improve their disease. However, treating both provides most effective therapeutic strategy ever seen this This combined returned near-normal life span dramatically improved quality life.