Voyager Therapeutics has announced the first patient dosed in RESTORE-1, a Phase II trial of VY-AADC gene therapy for the treatment of Parkinson’s disease.
Browsing: gene therapy
This week we look at gammaCore™ for cluster headache, warnings of stroke risk with a multiple sclerosis drug and a gene therapy for spinal muscular atrophy.
Gene therapy for disorders such as amyotrophic lateral sclerosis, Parkinson’s disease and Batten disease has seen success in animal models.
Researchers have revealed that reducing mutant huntingtin protein in the brain could restore cognitive and psychiatric impairments in mice.
Exploring the use of antisense therapy for neurodegenerative diseases: an interview with Frank Bennett
In this interview, Frank Bennett speaks to us about the work on IONIS-HTTRx in patients with early-stage Huntington’s disease and how this therapy could be applied to treat other neurodegenerative diseases.
This week’s industry news round-up reveals the latest updates around spinal muscular atrophy, Parkinson’s disease and acute stroke.
Sara Mole (UCL) discusses her work on Batten disease, as well as the universal challenges encountered when researching and treating rare neurological diseases.
Research presented at SfN’s Neuroscience 2017 has highlighted novel applications of genetic and cellular techniques that may help us to better understand neurological disorders such as addiction and Zika.
An international research team have shown that gene editing reverses Huntington’s symptoms without off-target effects in a mouse model.
Researchers from the University of Central Florida hope to use the CRISPR Cas9 method to screen drugs for Parkinson’s disease.