NSCFA/BrAsh-AT: Using derived liver organoids to test the efficacy of an existing therapeutic (CAT-SKL) to overcome or improve liver dysfunction in children with Ataxia Telangiectasia

A-T (also known as Louis–Bar syndrome) is a rare, genetic, progressive and life-limiting disease affecting multiple body systems resulting in ataxia, liver dysfunction, neuronal degeneration, immune deficiency, respiratory complications and increased cancer risks. This disease is caused by a mutation in the ATM gene and there are currently no effective therapies. The project is using derived liver organoids to test the efficacy of an existing therapeutic (CAT-SKL) to overcome or improve liver dysfunction in children with A-T.