Going blind in old age is a terrible affliction that kind hugely impact on the quality of life of the elderly and is unfortunately relatively common as a result of age-related macular degeneration. It is a progressive condition that can eventually lead to the complete loss of sight over the years.
A revolutionary new gene therapy could, however, prove to be a cure for macular degeneration. Not only saving millions from encroaching darkness as they age but even reversing macular degeneration in those already afflicted with severely limited sight verging on or already defined as blindness.
A one-time ‘genetic jab’ of a modified virus has, however, shown hugely positive results when administered to mice. And human trials will now begin, which, if as positive, could nullify the threat of age-related blindness for future generations.
The treatment uses a viral vector modified to continue a single gene. The gene hosted by the viral vehicle is central to the production of a protein that is central to how our eyes perceive light. When the viral vector travels into cells in our retina, the gene it contains catalyses these cells into producing the light sensitive protein. These cells then take on the job previously carried out by the photoreceptor cells that have stopped to function due to age-related wear and tear.
Source: The Times
Details of the revolutionary biotechnology treatment were yesterday published in the scientific journal Natural Gene Therapy. The researchers behind the study presented data on the recovery of sight in previously completely blind mice treated by the jab. Human trials have now already begun in India and are due to also start in the United States in November. The U.S. trials are being funded by the country’s National Institute of Health.
Samerendra Mohanty, a co-author of the study, shared that initial results from the human trials already underway have shown huge promise, with details to be made public at an upcoming conference of the American Academy of Ophthalmology. There is optimism that the treatment will allow people whose blindness has progressed to a point they are unable to count fingers held up four feet away to see their vision improved to the point they are able to comfortably read a book.
Dr Mohanty, chief scientific officer for Nanoscope Technologies, the U.S. biotech company behind the therapy, stated that the breakthrough could represent a “life-changing treatment”.
The gene that the treatment is based on is that which holds the code that allows cells in the retina to produce the light-sensory MCO1 protein. It is a molecule that somehow facilitates the rush of signals between cells that sight is based on. Health eyes contain cells called rod and cone photoreceptors in the retina, which produce MCO1, which is known as opsin.
These photoreceptors are activated by light and then send a signal through retinal neurons, into the optic nerve and ultimately to neurons firing in the brain. Damaged photoreceptors are behind several common eye diseases, including age-related macular degeneration and the genetic disorder retinitis pigmentosa.
As long as other retinal neurons, specifically the class of ‘bipolar cells’, that the new treatment activates into producing opsin instead of the usual photoreceptors, are intact, the therapy should be effective.
“The beauty of our strategy is its simplicity”, commented Dr Mohanty.