Ask A Scientist – Q&A with the Kids Neuroscience Centre

The Kids Neuroscience Centre, of the Sydney Children’s Hospital Network, is answering your questions! See the latest answer to a question from Justin below. If you’d like to ask a scientist a question, email it to

Question from Justin: I was just reading about stem cell research and wondered if there is any trials with any types of muscular dystrophy yet?

Answer from Michaela Yuen, PhD – Postdoctoral Research Fellow at Kids Neuroscience Centre:

“Stem cells and their use for the treatment of muscular dystrophies are a rather complex topic. There are a several different approaches to using stem cells as a treatment for DMD. Some strategies aim at fixing the underlying problem (e.g. stem cells help regenerate the muscle and restore dystrophin expression) while others appear to provide symptomatic relief (probably via anti-inflammatory action) similar to steroid treatments. Also, some treatments use cells from the body of the patient (autologous stem cell therapy), while others use cells from a healthy donor (heterologous stem cell therapy).

Have a look at the PDF for more information and resources on cell therapy and find out where the clinical research is up to.”