Gene therapy aimed at restoring sight for 30,000 people with Leber's congenital amaurosis.

Gene therapy aimed at restoring sight for 30,000 people - Guardian

Extract:

"British scientists are to launch the world's first clinical trials of a controversial gene therapy to cure childhood blindness.

Researchers will test the treatment on volunteers with a rare inherited form of blindness, in which a single defective gene causes the retina to degenerate and eventually stop working as the child grows up.

The condition, called Leber's congenital amaurosis, affects one in 80,000 in Britain. Children born with the defective gene are often completely blind by their 20s. If the trials are a success, it could revolutionise the treatment of more than 100 inherited forms of blindness that affect up to 30,000 people in Britain.

Patients taking part in the trial will be injected with a virus modified to carry a correct version of the faulty gene.

Once inside the eye, the virus ferries the healthy gene into the cells that make up the retina, halting and even reversing the damage caused.

The phase 1 trials are designed to assess the safety and efficacy of the treatment in 12 patients and will be carried out by the Institute of Ophthalmology at University College London and nearby Moorfields Eye Hospital. The safety of the treatment will be under particular scrutiny, following a gene therapy trial four years ago in which two children being treated for a rare immune disorder called Scid (severe combined immunodeficiency) developed leukaemia. Their cancers arose when a virus used to deliver healthy genes accidentally led to tumour-promoting genes."


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