Medical research

New therapy could treat children with Hunter disease

A team at the University of Manchester have developed a novel stem cell gene therapy approach to treat children with a devastating genetic disease. The approach is currently being developed for clinical trial in patients ...

Genetics

Gene therapy used to treat Fabry disease—a world first

A team of Canadian physicians and researchers is believed to be the first in the world to have used gene therapy to treat a patient with Fabry disease, a rare inherited enzyme deficiency that can damage major organs and shorten ...

Genetics

UK experts give green light to 'three-parent babies'

British scientists on Wednesday approved the use of so-called "three-parent baby" fertility treatments, paving the way for the country to become the first in the world to officially introduce the procedures.

Medical research

Attacking a rare disease at its source with gene therapy

Treating the rare disease MPS I is a challenge. MPS I, caused by the deficiency of a key enzyme called IDUA, eventually leads to the abnormal accumulation of certain molecules and cell death.

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