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What do new gene and cell therapies have to offer?

November 24, 2022

  • What do new gene and cell therapies have to offer conditions like cancer, inherited eye diseases, spinal muscular atrophy and other degenerative diseases?
  • Can we engineer immune cells to treat cancer?
  • Or bring eye sight by fixing a faulty gene?
  • Are inherited genetic diseases a life sentence?

The convergence of stem cell science and gene therapies are set to impact the future healthcare, with the potential to treat leukaemia, inherited eye diseases, muscular dystrophy, and more.

Gene therapy aims to treat disease by replacing faulty genes or introducing new genes. The field is still in its early days, but there are gene therapies in use, treating some blood cancers and eye conditions.

But what exactly is it? How are stem cells involved? What will it treat? And when? Experts answered these questions and more at the recent Gene therapies and stem cell research: now and in the future public forum.

A panel of researchers and clinicians sharing their perspectives on treatments for:

  • leukaemia and haemophilia: Dr Ashley Ng, WEHI, Peter MacCallum Cancer Centre and Royal Melbourne Hospital
  • childhood neurological diseases: Dr Sandi Kariyawasam, Sydney Children’s Hospital
  • retinal diseases: Dr Tom Edwards, Centre for Eye Research Australia
  • blindness and deafness: Dr Anai Gonzalez-Cordero, Children’s Medical Research Institute
  • muscular dystrophy: Dr Peter Houweling, Murdoch Children’s Research Institute

This forum was hosted by Foundation director and stem cell biologist Professor Megan Munsie. It also featured the moving story of advocate and community leader Julie Cini who lost both of her children to the rare genetic condition spinal muscular atrophy.

The event was hosted at the State Library of Victoria by the Australasian Society of Stem Cell Research. The Foundation supported the event as part of our mission to provide community education.

Watch a video recording of the event:

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