Novel, long-term treatment of Huntington’s disease by gene therapy
Novel synthetic zinc finger proteins delivered to the brain through the use of viral vectors, efficiently and selectively repress the mutant huntingtin gene in a mouse model of Huntington’s disease long term after a single intervention, and successfully restore the motor coordination to levels comparable to those in healthy controls in an acute model...
Inventor(s): Mark Isalan
Keywords(s): Biotechnology, Gene Therapy, Genetic Diseases, Neurodegenerative Diseases, Neuromuscular & Movement Disorders, Neuroscience, Rare / Orphan Diseases
Category(s): Biotechnology, General Biology & Chemistry, Therapeutics (Biologics & Compounds)