StemCell Therapy

Neveah Taouk, 4

At last, when Mary was seven and Neveah three, new developments in whole-genome sequencing enabled specialists to identify the disorder. The diagnosis gave the Taouks information but not hope. They knew what the problem was, but there was no treatment and no cure.

Desperate, Charlie contacted specialists around the world. I must have spoken to at least fifty people scientists, doctors, professors, he says. Most of them had never heard of the condition.

His search eventually led to Dr Wendy Gold, a specialist in rare genetic disorders in children, based at the University of Sydney and the Childrens Hospital at Westmead. We arranged to talk, says Charlie. To be honest, I wasnt expecting much. But then she said, Have you heard of gene therapy?

Gene therapy is a new and rapidly evolving field of research. One of the therapys forms involves adding new genes to a patients cells to replace missing or malfunctioning genes. The new genes are typically delivered to the appropriate cells in the body using a benign virus as a carrier. Gene therapy is already being used to treat diseases including spinal muscular atrophy. It could also be a promising treatment for Parkinsons disease. Dr Gold believed there was a chance it could help the Taouk girls.

Read the rest here:
The gene therapy research that could save a family of four - News - The University of Sydney

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