In a first, scientists attempt to halt blindness through gene therapy

Hitting a milestone, for the first time ever researchers have performed gene therapy operation in order to halt spreading of age-related blindness.

Scientists at the University of Oxford have performed the first gene therapy operation in order to stop expansion of the most common cause of vision loss known as age-related macular degeneration (AMD), with just a single injection.

The team carried out the first trial on an 80-year-old patient Janet Osborne. Osborne suffered from the condition in both eyes that lead to inability in performing daily chores, reported BBC. “We are harnessing the power of the virus, a naturally occurring organism, to deliver the DNA into the patient’s cells,” said lead researcher Robert MacLaren.

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The operation involved detaching the retina and injecting a solution containing a virus underneath. The virus contains modified DNA sequence that infects cells known as the retinal pigment epithelium (RPE), and corrects a genetic defect that causes AMD.

Though the therapy does not reverse the damage already done, but hopes to rapidly halt the disease’s progression and preserve what vision is left. Ideally, if the procedure is successful, it would only be required to perform once since the effects are thought to be long-lasting.

MacLaren explained that when the virus opens up inside the retinal cell, it releases the DNA of the gene cloned by the team. The cell then starts making a protein that is believed to modify the disease, correcting the ‘imbalance of the inflammation caused by the complement system’ – a system of proteins in immune system that fights bacteria, reported Daily Mail.

“The idea of this gene therapy is to ‘deactivate’ the complement system, but at a very specific point at the back of the eye, so the patient would otherwise be unaffected by it, and we hope that in future it will slow down the progression of AMD,” said McLaren.

If proved to be successful, it is hoped that gene therapy could have beneficial impact on patients’ life and their ability to remain independent. However, more trials are needed to mark the therapy as completely safe for clinical use.

“A genetic treatment administered early on to preserve the vision in patients who would otherwise lose their sight would be a tremendous breakthrough and certainly something I hope to see in the near future,” MacLaren expressed.