Novel gene editing approach to increase BDNF expression for neurological diseases

Applications: Pharmaceuticals

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.

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

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Novel gene editing approach to increase BDNF expression for neurological diseases

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