Gene Therapy In Eye Disease

This is something I call as “In the search of God”. The broad basic is replacing a defective gene that causes a disease by a healthy gene through an innocent virus. The eye being more avascular, the less is the immune response to new particle. So, there is progress in gene therapy. Please read the original article in BBC or The Lancet.
clipped from news.bbc.co.uk

Gene therapy can be particularly effective in treating inherited sight problems in children, fresh trials show.

Treatment so far has focused on Leber’s congenital amaurosis (LCA), a rare inherited disorder which causes gradual deterioration in vision and can lead to blindness by the time the patient is 20.

There were measurable improvements, including an at least 100-fold increase in pupillary light response – when the pupils constrict in brightness.

The genes are contained in a harmless virus which is unlikely to be attacked by the body as the immune system is not strong in the retina.

Gene therapy has already been successfully deployed to treat children with X-SCID – a life-threatening immune deficiency also known as “boy in the bubble syndrome”.

But a lot more research was needed, she added, “to maximise the benefits of gene therapy techniques and understand how they can then be turned into effective treatments for a variety of more common degenerative eye conditions.”

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