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...
20:11 , Aug 1, 2018 |  BC Innovations  |  Distillery Techniques

Drug platforms

TECHNOLOGY: Gene therapy A method enabling BBB entry, and temporal and cell type-specific control of chemogenetic therapies could be used to develop treatments for CNS diseases. The method, dubbed acoustically targeted chemogenetics (ATAC), involves three...
20:09 , Aug 1, 2018 |  BC Innovations  |  Distillery Therapeutics

Autoimmune disease; renal

INDICATION: Lupus; renal damage Patient sample and mouse studies suggest inhibiting CAMK4 could help treat lupus nephritis, focal segmental glomerulosclerosis (FSGS) and other podocytopathies. In kidney samples from lupus nephritis and FSGS patients, podocyte levels...
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...
18:22 , Jul 26, 2018 |  BC Innovations  |  Translation in Brief

A plasticity problem

New research from the University of Bordeaux suggests the synaptic pathology underlying Alzheimer’s disease is driven by aberrant activation of CAMK2 by β-amyloid oligomers. The findings may point to the missing link in how β-amyloid...
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...
20:01 , Feb 1, 2018 |  BC Innovations  |  Translation in Brief

New Therapeutic Targets and Biomarkers: January 2018

New Therapeutic Targets and Biomarkers: January 2018 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during January 2018. Therapeutic targets are defined as any protein, gene or other...
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...