Is Cure for Inherited Retinal Disorders Finally in the Cards?

by Reshma Anand on April 29, 2016 at 2:19 PM

A gene therapy has restored vision in people with a rare form of genetic blindness, retaining the eyesight for up to four years.

Oxford University researchers developed the breakthrough technique and they hope that the therapy may be a potential cure for common vision loss and genetic-related visual impairments in people.

Gene Therapy Platform for Macular Degeneration Patients

Millions of adults over age 50 struggle each year with vision loss caused by damage to the retina or common macular degeneration.

‘Gene therapy may be a potential cure for common vision loss and genetic-related visual impairments in people.’

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Is Cure for Inherited Retinal Disorders Finally in the Cards?

The gene therapy was applied to six patients suffering from choroideremia, which is caused by a defect in the gene CHM and affects about one in 50,000 people in the UK. There is currently no cure for this disease and as the disease progresses, it damages the retinal photoreceptors.

Gene therapy involves the process of using the infectious properties of a virus to insert DNA into a cell, where the viral DNA is replaced with DNA that is reprogrammed to correct whichever gene is faulty in the patient.

Gene Editing Technique Offers Precised Blindness Therapy by Repairing Stem Cells

CRISPR/Cas9 has been heralded as a major breakthrough in genetic engineering to precisely make specific alterations to the DNA in cells and experimental animals.

The patients were directly injected a harmless virus containing the healthy CHM gene in the retinal cells. After closely following them for four years they found the following results,

Two patients had an improvement in their eyesight which lasted for four years
Three patients retained their vision and had no gradual loss
One patient who was given lower dose of the gene experienced a decline in their eyesight

This showed that injecting a virus to deliver healthy genes to replace the missing gene prevented loss of vision in people suffering from the genetic disorder. This positive results increased the hopes of treating other rare genetic disorders such as retinitis pigmentosa.

Lead Investigator of the Study, Robert MacLaren, said, "In this case, success in getting a treatment effect that lasts at least several years was achieved because the viral DNA had an optimal design and the viral vector was delivered into the correct place, using advanced surgical techniques. In brief, this is the breakthrough we have all been waiting for."

Source: Medindia

Inherited Blindness Can be Treated Using Gene-Editing Technique

Retinitis pigmentosa is a class of diseases in which patients experience night blindness in the early stages, along with atrophy and pigment changes in the retina.

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