Talk
Characterising disease relevant signatures in iPSC-derived motor neurons to test the therapeutic potential of homeoprotein EN1
Discover how Dr Di Lullo, BrainEver, is studying disease phenotypes and the potential of homeoprotein Engrailed 1 as a novel therapeutic for ALS, using patient-derived motor neurons with TDP43 mutations.
Discover how Dr Di Lullo, BrainEver, is studying disease phenotypes and the potential of homeoprotein Engrailed 1 as a novel therapeutic for ALS, using patient-derived motor neurons with TDP43 mutations.
Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease characterised by motor neuron degeneration. The development of robust models of ALS remains a significant challenge for drug development and preclinical validation studies. BrainEver is a preclinical stage biotech targeting ALS with an innovative homeoprotein therapy approach. In vivo studies using a mouse model with ALS-like phenotypes has demonstrated a strong therapeutic potential for the homeoprotein Engrailed 1 in ALS. The use of patient-derived iPSCs and subsequent differentiation into motor neurons represents a unique opportunity to recapitulate disease phenotypes and test disease-modifying drugs. Modelling TDP43 pathology is of notable interest due to its observed presence in both familial and sporadic forms of ALS. BrainEver has been investigating if ²»Á¼Ñо¿Ëù ioMotor Neurons harbouring a TDP43 mutation demonstrate disease-associated mutations such as TDP43 mis-localisation, TDP43 aggregation and the expression of cryptic exons which can serve downstream for drug development assays.