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Retinal gene therapy

Kumaran, N; Michaelides, M; Smith, AJ; Ali, RR; Bainbridge, JWB; (2018) Retinal gene therapy. British Medical Bulletin , 126 (1) pp. 13-25. 10.1093/bmb/ldy005. Green open access

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Abstract

INTRODUCTION: Inherited retinal diseases are the leading cause of sight impairment in people of working age in England and Wales, and the second commonest in childhood. Gene therapy offers the potential for benefit. SOURCES OF DATA: Pubmed and clinicaltrials.gov. Areas of agreement: Gene therapy can improve vision in RPE65-associated Leber Congenital Amaurosis (RPE65-LCA). Potential benefit depends on efficient gene transfer and is limited by the extent of retinal degeneration. AREAS OF CONTROVERSY: The magnitude of vision improvement from RPE65-LCA gene therapy is suboptimal, and its durability may be limited by progressive retinal degeneration. GROWING POINTS: The safety and potential benefit of gene therapy for inherited and acquired retinal diseases is being explored in a rapidly expanding number of trials. Areas timely for developing research: Developments in vector design and delivery will enable greater efficiency and safety of gene transfer. Optimization of trial design will accelerate reliable assessment of outcomes.

Type: Article
Title: Retinal gene therapy
Location: England
Open access status: An open access version is available from UCL Discovery
DOI: 10.1093/bmb/ldy005
Publisher version: http://dx.doi.org/10.1093/bmb/ldy005
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: gene therapy, retina, retinal diseases, gene therapy, retinal diseases, vision, retina
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > Institute of Ophthalmology
URI: https://discovery.ucl.ac.uk/id/eprint/10047156
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