BioCentury | Jun 26, 2020
Distillery Therapeutics

FMR1 peptide therapy for Fragile X syndrome

DISEASE CATEGORY: Neurology INDICATION: Fragile X syndrome An FMR1-based peptide therapy could treat Fragile X syndrome. The therapy comprises a FMR1 N-terminal fragment -- the first 297 residues -- conjugated to a cell-penetrating peptide derived...
BC Innovations | May 15, 2019
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...
BC Innovations | May 6, 2019
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...
BC Innovations | Oct 31, 2018
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...
BC Innovations | Jul 31, 2018
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...
BC Innovations | Jun 6, 2018
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...
BC Innovations | Jan 23, 2018
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...
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