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First gene therapy for a rare eye disease, now NHS-approved

This life-changing new gene therapy will halt sight loss and improve vision

17-Feb-2020

Key points from article :

Luxturna (voretigene neparvovec, for retina treatment, has been approved by NICE.

With less than 90 people eligible for treatment in England.

For those with retinal dystrophy due to inheriting faulty RPE65 gene from both parents.

Injection is made into back of the eye which delivers working copies of RPE65 gene.

Contained inside a harmless virus, enables them to penetrate the retinal cells.

Gene provides instructions to make RPE65 protein, essential for healthy vision.

Treatment is only suitable for patients who have some remaining vision.

Unknown yet how long the benefits will last, it could be several decades.

Mentioned in this article:

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James Bainbridge

Consultant Ophthalmic Surgeon-Scientist, Moorfields Eye Hospital, Chair of Retinal Studies, UCL

John Radcliffe Hospital

Oxfordshire's main accident and emergency site

Luxturna (voretigene neparvovec)

Prescription gene therapy product for treatment of inherited retinal disease

Moorfields Eye Hospital London

One of the world’s leading eye hospitals, providing expertise in clinical care, research and teaching and education

National Institute for Health and Care Excellence (NICE)

This institution provides national guidance and advice to improve health and social care

NHS

UK National Health Service, publicly funded healthcare system in England

Novartis

Global medicines company for developing and producing drugs.

Robert MacLaren

Professor of Ophthalmology at University of Oxford.

Stephen Powis

National Medical Director of NHS England and Professor of Renal Medicine.

Topics mentioned on this page:
Vision (health), Gene Therapy