LONDON: 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. 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. 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.
“The idea of this gene therapy is to ‘deactivate’ the complement system, 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.
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.