Researchers at Oxford University are very excited about a new gene therapy targeting retinitis pigmentosa. Over the next few months, British doctors hope to test out this new treatment method on up to 30 patients from the UK.
Oxford researchers involved in this study first tested out their gene therapy treatment on a 29-year-old British male patient. The procedure took place on March 14th. Everyone involved in the study said the therapy went well and the patient is now resting at home with his family.
Since retinitis pigmentosa is an x-linked genetic disorder, it is often referred to as XLRP for short. There's currently no cure for this disease that affects approximately 15,000 people in the UK.
Many people with XLRP notice a narrowing of vision in their teens or early 20s. A few other symptoms include blurred vision and the inability to see in darkened rooms. Although XLRP is rather rare, it is the leading cause for early blindness in all developed nations.
Professor Robert McLaren, who teaches ophthalmology at Oxford, is the head researcher on this study. All of these eye operations are being conducted at the Oxford Eye Hospital in Oxford's John Radcliffe Hospital.
When asked how this therapy works to correct XLRP, Dr. MacLaren told reporters that his doctors use the latest in gene technology to both develop and implant a corrective strand of DNA into the patient's retina. This DNA is placed in a person's eye using a harmless virus.
It will take a few years of follow-ups to see just how safe this new therapy is on all the patients who choose to participate in the study. While doctors will also study this therapy's overall effectiveness, their main focus in this study is on the therapy's safety.
The main gene that goes haywire in people with retinitis pigmentosa is known as the RPGR. In healthy individuals, RPGR is responsible for moving proteins safely through the eye to produce optimal vision. Unfortunately, the people with retinitis pigmentosa have a faulty RPGR that wreaks havoc on the retinae and often causes blindness at a young age.
Researchers had to develop a gene that would balance out this faulty RPGR. People involved in developing the gene therapy said that it was extremely difficult coming up with just the right DNA code to stabilize the patient's faulty RPGR.
Professor MacLaren told reporters he is hopeful this new therapy will help people affected by this disease regain their sight and independence. Although it will take awhile to gather all the data on this new gene therapy, he believes his team is onto something truly revolutionary in the field of eye care.