主讲人：PROFESSOR JOHN RASKO, AO
讲座时间：10:00 — 11:30
题目：Molecular medicine - Gene therapy is now
报告人：PROFESSOR JOHN RASKO, AO
Professor, Sydney Medical School, University of Sydney
Head, Gene and Stem Cell Therapy Program, Centenary Institute
Head of Department, Cell & Molecular Therapies, Royal Prince Alfred Hospital, Sydney AUSTRALIA
The field of genetics originally provided insights into disease pathogenesis. More recently genomic medicine has facilitated improved prognostication, reproductive choices and therapeutic drug options. The challenge of realising the full potential of genetic understanding has been in overcoming the hurdles of efficient gene therapy. Since the first human clinical trial using gene technology in 1989, there have been over 2400 approved clinical trials worldwide. A few dozen clinical trails have been undertaken in Australia representing just over 1% of the trials worldwide. The overwhelming majority of human clinical trials involves short-term gene expression or random integration of a therapeutic gene. Emerging technologies require controlled development in compliance with safety, regulatory and GMP requirements.
More precise gene targeting tools were first described in the early 2000s. Targeted gene editing or replacement using Zinc Finger Nucleases or TALENS have been tested in about a dozen clinical trials since 2009. These include attempts to delete the CCR5 protein on T cells (completed 2015+) and therapeutic ZFN-mediated genome editing in mucopolysaccharidosis (recruiting 2016+) and the haemophilias (recruiting 2016+). The pace of clinical development has accelerated over nearly three decades of gene therapy. However within this context, its worth noting that the first ever (controversial) use of CRISPR to delete PD-1 in a lung cancer patient was administered in October 2016. Highlights in the clinical gene therapy field will be discussed with special reference to haemophilia, thalassemia, blindness and cancer.