IB PRIME COMPETITION (OCTOBER 2018) WINNING PROJECT: SYNPROMICS AND UNIVERSITY OF EDINBURGH

CNS-specific promoters for gene therapy applications

Central Nervous System (CNS) diseases such as Alzheimer’s, Parkinson’s and ALS cause significant impact to the affected individual, their family and society at large. Adeno-associated-virus (AAV) gene-therapy represents a powerful tool for the treatment of these neurodegenerative and other CNS-related diseases. A therapeutic gene is delivered by AAV, which corrects a specific defective gene. A challenge of gene-therapy treatment development is the target cell type specific expression of the therapeutic gene at a physiologically appropriate level. 

We will discover and dissect brain-specific gene regulatory elements to engineer CNS cell-specific gene promoters. CNS cell type activity will be determined using cutting edge single-cell sequencing approach followed further characterisation of specific brain regions. This allows the identification of promising candidates, which can be utilised in a machine learning approach where regulatory elements will be used to generate novel, synthetic and cell-type-specific promoters to further develop gene-therapy treatments for neurological diseases.

Industry Partner: Synpromics

Academic Partner: University of Edinburgh - Matt Nolan

IBioIC Funding: £49,197.00

Total Project Cost: £250,782.00