Gene therapy hope for childhood blindness
Researchers at University College London are the first in the world to attempt to use gene therapy to treat a rare inherited condition which leads to blindness in childhood. The disease, known as Leber Congenital Syndrome (LCS), is caused by an abnormality in the RPE65 gene, which...
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Miracle Cure or Media Hype?
by Celine Lewis
Last week's BioNews reported on the development of new drug called PTC124, which offers hope to patients with Duchenne muscular dystrophy (DMD), as well as cystic fibrosis (CF) and perhaps a number of other genetic disorders. The drug is currently at the stage of clinical trials after successful trials on...