Sara Mole (UCL) discusses her work on Batten disease, as well as the universal challenges encountered when researching and treating rare neurological diseases.
Browsing: gene therapy
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.
Researchers report the restoration of higher levels of hearing – the equivalent of a whisper – in genetically deaf mice using a gene therapy vector.
Researchers demonstrate that amyotrophic lateral sclerosis (ALS) neuronal degeneration may be reduced following treatment with a growth factor, IGF-2, indicating a possible new target for ALS treatment.