A first of its kind brain organoid grown at The University of Queensland has helped researchers identify therapies that reduce the impact of COVID-19 on people with Down syndrome.
For three months Tahmina Tabassum relearned what she knew about industry and entrepreneurship during a placement with biotech Azafaros BV, a whirlwind experience that included a stint at the company’s headquarters in Basel.
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.