Chief Investigator: Professor Jozef Gecz Funding Amount: $ 100,000 Recipient: The University of Adelaide Overview: One in six children has disability related to brain function. The most severe neuro-disabilities are predominantly genetic and can be passed on from generation to generation. We have identified a severe, childhood-lethal genetic disorder of the brain and muscles, very … Continue reading Oligonucleotide-driven treatment of an inherited, childhood-lethal polyneuropathy: Generation and characterisation of a humanised preclinical mouse model