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Genea Biocells creates human skeletal muscle cells to aid treatment of Muscular Dystrophy

Genea Biocells creates human skeletal muscle cells to aid treatment of Muscular Dystrophy

SYDNEY, 10 October 2013: Scientists at Genea Biocells have achieved an Australian first by producing human skeletal muscle from stem cells – a breakthrough expected to aid in the treatment of muscular dystrophies.

The discovery of a technique to turn human embryonic stem (hES) cells into skeletal muscle cells in a consistent, scalable and highly efficient manner gives researchers an invaluable tool to better understand the mechanisms of muscle diseases. It is expected to ultimately lead to the discovery and development of effective treatments for muscular dystrophies, a diverse group of debilitating genetic diseases.

“This is the first time in Australia that differentiation of human skeletal muscle from pluripotent stem cells has been achieved and the first time in the world that it has been done with significant yields and without cell sorting or genetic manipulation,” Genea Biocells General Manager Dr Uli Schmidt said.

“Our muscle project team, led by Senior Scientist Dr Leslie Caron, undertook a technically challenging task that had never been successfully achieved on this scale before – turning hES cells into the skeletal muscles that allow us to move and smile.

“This world first breakthrough, developed with the support of Australian not-for-profit organisation FSHD Global Research Foundation, means we can produce disease affected human skeletal muscle cells with broad applications in modelling muscular diseases and therefore developing treatments.”

Muscular dystrophies are a diverse group of genetic disorders, characterised by weakness and atrophy of various voluntary muscles in the body. Facioscapulohumeral Dystrophy (FSHD) is one of the most common forms of muscular dystrophy and is thought to affect seven in every 100,000 people. It is a progressive disorder which can rob sufferers of the ability to smile and talk and eventually to walk.

“The FSHD Global Research Foundation is committed to finding a cure or treatment for FSHD and this discovery may take us one step closer to that goal. It is critical that our research foundation continues to raise money and raise awareness about the disease so that we can keep funding world leading researchers like Genea,” FSHD Global Chairman and founder Bill Moss said.

Before the launch of FSHD Global in 2007, there had been no focused research or funding for FSHD research in Australia and very little internationally, leaving research into FSHD 20 years behind other forms of muscular dystrophy. Little is known about the cause of this disease and no treatment is currently available.

This discovery was made possible by the awarding of the Bill Moss Fellowship to Dr Caron.

“The real world application of this discovery will be human myoblasts, the immediate precursors to skeletal muscle cells, supplied as strictly quality controlled frozen stocks for biotechnology and pharmaceutical companies to use in drug discovery for FSHD. Put simply, this discovery provides a tool which will speed up drug development,” Dr Schmidt said.

For more information about Genea FSHD pipeline.

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