Data-driven drug discovery for A-T – interrogation of dysfunctional pathways at single-cell resolution in cerebellar organoids
Action for A-T
QIMR Berghofer Medical Research Institute
Using a data-driven approach, informed by single-cell sequencing of a large cohort of ATM_x0002_deficient cerebellar brain organoids - the hypothsis is that computational prediction of drugs will allow selective, positive modulation of aberrant pathways and behaviour in the team's in vitro model of the A-T cerebellum. Further, the team will democratize this research space by sharing their data, placing the building blocks for expansion of this project to grow globally.
1.1 Normal biological development and functioning