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Gene Therapy at your Doorstep

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Times of India
5 February, 2010
Bangalore, India

Brings Ray Of Hope To Blind Children Narayana Nethralaya Leads The Way
Gene Therapy
Future of medicine lies in gene therapy, which now comes as a blessing to the visually impaired in Bangalore. Research and screening for genetic eye disorders will soon begin in the city – and leading the medical initiative will be Narayana Nethralaya.

Using frontline genetic tools, it will be possible to catch early footprints of eye diseases that cause blindness in children. Treatment is most effective when a disease is caught at the onset.

Making these options available in Bangalore is a team of pioneer researchers from London, who are sharing their experience and work with Indian doctors.

Dr Rohit Shetty, head of research, Narayana Nethralaya, said: “Ten per cent of blindness is due to gene abnormalities and 60% of it is in children. The future of treatment will be directed at identifying the causative abnormal genes and correcting them using gene therapy. Narayana Nethralaya will bring this treatment to India soon.”

While gene therapy has been debated the world over as a future medicine, it is now a reality in select diseases of the eye, especially children. Ophthalmology is the first field in medicine where genetic diagnosis and therapy have made big strides, especially in certain groups of eye diseases caused by a genetic aberration. The therapy has been tried in a disease called Leber Congenital Amaurosis (LCA) – a rare genetic disorder in which retinal dysfunction causes vision loss, often from birth.

Prof Helene Dollfus from France says: “It is important to tell people that complexity of these disorders is huge, and research is warranted to find therapies. Not all genes are known yet and not all the cellular mechanisms leading to the retinal degeneration have been disentangled. However, huge progress has emerged with gene therapy developed by innovative laboratories.”

It can Happen in Bangalore
Said Prof Michael J Denton from New Zealand: “There has been a significant breakthrough for patients with a defect in one of the genes – called RPE65 – which nourishes the retina and functions in the epithelial cells lining the back of the retina. They have experienced an improved vision after receiving injections in the back of the eye.” “For me, the news is particularly gratifying as the gene was first identified in a family from Bangalore. It was collected for gene mapping studies by myself and Prof Kumaramanickavel in the mid–1990s, while we were working together on inherited retinal diseases.” The researchers were in Bangalore to attend a twoday global conference on recent advances in genetics and gene therapy, hosted at Narayana Nethralaya.

Tie–up with UCL
Narayana Nethralaya will collaborate with University College of London (UCL) on gene therapy. We will exchange doctors in the coming three months, so there is effective information sharing. Screening of patients with Leber Congenital Amaurosus will be done at Narayana Nethralaya and subsequently, provision for trials will also be put in place.

By Dr G Kumaramanickavel
Director of Research, Narayana Nethralaya

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