We will use hiPSC-derived brain organoids and screen a library of approved drugs to identify those able to alter neural activity in a patient-specific model, train an artificial intelligence model that leverages both clinical and genomic data to assist drug selection, and validate treatment predictions in real-world clinical settings.
While we know that DNA mutations in the tRNA synthase gene DARS cause the pathology, the precise molecular and cellular processes that underlie this leukodystrophy remain unclear and no effective therapeutics exist.
This consumer-driven project will leverage brain organoid models derived from hIPSC lines from patients with the genetic hereditary spastic paraplegia SPG56 to test the safety and efficacy of viral gene delivery therapy.
Ataxia-Telangiectasia (A-T) is caused by mutations in the ATM kinase, a protein involved in DNA break repair, oxidative stress regulation and metabolism.
Replacing a defective gene via AAV mediated gene therapy is a powerful therapeutic approach. This project will develop and test safer gene therapy approaches in pre-clinical human brain organoid models.
For those interested in: stem cell ageing and regenerative engineering
Human brain development can be severely impacted by a lack of oxygen perinatally. This project will study the molecular and cellular processes that are responsible in human stem cell derived brain organoid models.
For those interested in: Stem cell ageing and regenerative engineering
