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Manchester Doctors Pilot New Gene Therapy Treatment For Retinitis Pigmentosa

17.11.2017

British eye doctors have begun using a new gene therapy procedure on patients with X-linked retinitis pigmentosa (XLRP). Ophthalmologists around the world are hopeful this new procedure can be used to successfully treat XLRP patients in the future.

This pilot gene therapy procedure is taking place at the Manchester Royal Eye Hospital (MREH). Doctors involved in this study modified the retinitis pigmentosa GTPase regulator (RPGR) gene and injected the serum into 24 XLRP patients.

Geneticists believe the RPGR gene is responsible for both making and transporting proteins in the eyes that are necessary for normal sight. People who have XLRP have a malfunction in the RPGR gene.

Many prominent UK institutions are supporting this trial. Both Saint Mary's Hospital and the NIHR Manchester Clinical Research Facility have made significant contributions to MREH's research. Also, the London-based biopharmaceutical company Nightstar is the study's main sponsor.

Dr. Paulo Stanga is the head researcher on this trial. Mr. Stanga works as a consultant ophthalmologist and a vitreoretinal surgeon at MREH.

When asked what he thought of this pilot test, Dr. Stanga told reporters, "We're delighted to be able to offer our patients the opportunity to participate in this trial for this new treatment for X-linked retinitis pigmentosa." Dr. Stanga also said he believes this gene therapy has the potential to cure other retina-related eye diseases.

XLRP is an inherited disorder which doctors believe travels through the RPGR gene. Although rare, retinitis pigmentosa can easily lead to permanent blindness if it's not diagnosed early on. As the name suggests, retinitis pigmentosa adversely affects a person's retina.

Symptoms of retinitis pigmentosa are usually first seen in early childhood. These symptoms include tunnel vision, inability to see in dimly lit areas, and blurry vision. In the UK, doctors estimate that 1 in 3,000 people have retinitis pigmentosa.

There's no official treatment for retinitis pigmentosa as of today. All eye doctors, however, recommend retinitis pigmentosa patients wear sunglasses to prevent further damage to the eyes.

The company Nightstar Therapeutics is dedicated to finding new ways to treat blinding diseases. Nightstar has its main headquarters on 215 Euston Road in London and a secondary office in Lexington, Massachusetts.

W. J. Wilson founded the MREH way back in 1814. Despite being bombed by the Germans during World War II, this hospital is still standing and remains is one of Great Britain's most prestigious ophthalmic hospitals. MREH is on Hathersage Road right next to the Manchester Royal Infirmary and Saint Mary's Hospital.






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