Novel gene editing approach to increase BDNF expression for neurological diseases
Gene editing of BDNF to treat neurological disease.
Applications: Pharmaceuticals
| Features | Benefits |
|---|---|
| Targets a brain relevant isoform of BDNF | Focussed editing for neurological conditions |
| Generates 1.5-3 fold increase in BDNF | Physiological levels of increase, reducing effects of over-expression |
| Editing does not alter native promoter, enhancers, and splicing | Ensures tissue specificity, and temporal and activity-dependent control |
| Specific base editing | Does not require delivery of large editing machinery |
Patented and Available For
- Co-development
- Licensing
Brain-derived neurotrophic factor (BDNF) is a key regulator of neuronal survival and synaptic function, and reduced BDNF signalling is implicated in a broad range of neurodegenerative and neurological indications, including Alzheimer’s, Parkinson’s, ALS, and stroke.
This invention targets a specific uORF start codon in a brain-relevant BDNF isoform to generate modest increases in expression to levels that show efficacy in pre-clinical models, whilst maintaining the native cellular control.