AICC(WA) Murdoch University Science and Society event: "Personalised genetic medicines: hopes and hype"

Featuring - Professor Steve Wilton, Foundation Chairman in Molecular Therapy, Centre for Comparative Genomics, Murdoch University, Professor Sue Fletcher, Principal Research Fellow, Centre for Comparative Genomics, Murdoch University and Moderator: "Naked Scientist" Dr Chris Smith, Cambridge University.
“Personalized genetic medicines: hopes and hype”

Personalized medicine is getting the right drug to the right person or, sometimes more importantly, ensuring someone is not administered something that could cause an adverse reaction. With the sequencing of the human genome and elucidation of our “normal” genetic blueprints, it is now possible to identify variations in genes that may cause rare diseases (muscular dystrophy, cystic fibrosis), predispose an individual to one of the more common and complex conditions (multiple sclerosis, asthma, Alzheimer’s) or cause a severe and life-threatening adverse drug reaction. Genetic medicines are being developed to treat serious inherited conditions that are caused by single gene mutations. One gene drug, Eteplirsen, has been designed to re-program processing of a defective dystrophin gene message, and in extended clinical trials shows that the relentless progression of muscle wasting seen in Duchenne muscular dystrophy is halted. Proof-of-concept has been demonstrated that we can rescue functional gene expression despite catastrophic defects in crucial nerve and muscle genes. Can we extend this technology to other genetic conditions or predispositions, essentially customizing the treatment to each patient’s unique genetic blueprint?