"...Neurotrophic factors may be the key to the cure for Parkinson’s, Huntington’s, Alzheimer’s, and other neurodegenerative disorders. Scientists have known this for over twenty years. But the question continues to loom – how does one safely and effectively deliver the neurotrophic factors to the damaged neurons? Dr. Raymond Bartus and his team at Ceregene, a biotechnology company in San Diego, have developed an innovative approach that may be the answer.
Rather than focusing on conventional methods of neurotrophic factor delivery, which have always been extremely difficult and resulted in undesirable side effects, the Ceregene researchers took a different approach. They turned to gene therapy. Instead of delivering the restorative protein to the targeted sites in the brain, the Ceregene researchers developed a way to deliver only the gene for the protein. Once in place, the gene induces local cells to make the protein on site.
“[CERE-120] is designed to deliver the gene for NRTN to targeted neurons, and subsequently program these neurons to provide continuous, long-term, predictable NRTN expression in selective, stereotactically-targeted regions of the brain,” say the authors in their paper published in the Neurobiology of Aging..."