16:51 , May 15, 2019 |  BC Innovations  |  Translation in Brief

Lentiviral gene therapy could offer relief to refractory epilepsy patients

Within a year, University College London researchers hope to begin a clinical trial of a gene therapy for epilepsy, a non-monogenic disorder. The hope is that the approach will offer an alternative to brain surgery...
19:19 , May 6, 2019 |  BC Innovations  |  Distillery Therapeutics

KCNA1 gene therapy for epilepsy

DISEASE CATEGORY: Neurology INDICATION: Epilepsy Rat studies suggest KCNA1-based gene therapy could help treat epilepsy. The gene therapy consisted of KCNA1 codon-optimized for expression in human cells and harboring the I400V mutation to increase the...
20:23 , Oct 31, 2018 |  BC Innovations  |  Distillery Therapeutics

Neurology

INDICATION: Addiction Rat studies suggest M1 muscarinic receptor (CHRM1)-based chemogenetic inhibition of the prelimbic paraventricular thalamic nucleus pathway could help treat cocaine addiction. The system for chemogenetically inhibiting the pathway is initiated by bilateral injection...
21:18 , Jul 31, 2018 |  BC Innovations  |  Translation in Brief

ATAC of the DREADDs

Scientists from the California Institute of Technology have combined ultrasound waves with gene and drug therapies to selectively and non-invasively control neural circuits. The approach could be used to treat neurologic and psychiatric diseases and...
19:04 , Jun 6, 2018 |  BC Innovations  |  Distillery Techniques

Biomarkers

TECHNOLOGY: Gene profiling A mutation in CAMK2A could help predict the risk of a previously undescribed neurodevelopmental disorder characterized by growth delay, frequent seizures and severe cognitive dysfunction. Genomic profiling of two children with the...
21:18 , Jan 23, 2018 |  BC Innovations  |  Distillery Therapeutics

Autoimmune disease; neurology

INDICATION: Multiple sclerosis (MS); pain Mouse studies suggest inhibiting CAMK2A could help treat MS, including MS-associated pain. In the experimental autoimmune encephalomyelitis (EAE) mouse model of MS, knock-in of an autophosphorylation-deficient CAMK2A variant or intrathecal...