More Powerful Gene Vector Aims To Restore Vision In Inherited Blindness

Professor James Bainbridge, MA, PhD, FRCOphthProfessor of Retinal Studies, UCL Institute of Ophthalmology NIHR Research Professor, NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of OphthalmologyMedicalResearch.com Interview with:
Professor James Bainbridge,
MA, PhD, FRCOphth
Professor of Retinal Studies, UCL Institute of Ophthalmology
NIHR Research Professor, NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of OphthalmologyConsultant Ophthalmologist, Moorfields Eye Hospital NHS Foundation Trust

Medical Research: What is the background for this study? What are the main findings?

Prof. Bainbridge : Leber Congenital Amaurosis (LCA) is one of the most common causes of inherited, untreatable blindness in children. There are at least 14 different types of Leber Congenital Amaurosis of which LCA Type 2 (LCA2), caused by defects in the gene RPE65, affects around one in 100,000 people worldwide. Evidence from animal studies support that LCA2 may be amenable to treatment with RPE65 gene replacement therapy.

The main findings of this phase I/II clinical trial confirm our preliminary findings (published in NEJM, 2008) that gene therapy can improve night vision. Improvements peak within the first 12 months after treatment but then decline during the three-year follow-up period which is consistent with the published results and interim findings from other studies of RPE65 gene therapy.

Medical Research: What should clinicians and patients take away from your report?

Prof. Bainbridge : This trial provides further evidence of benefit and safety for gene therapy in inherited blindness. However our data, together with results of a parallel study in dogs, indicate that the demand for RPE65 is not fully met with the current generation of gene therapies. We have concluded that early intervention using a more potent gene therapy vector, expressing higher levels of RPE65 is likely to provide greater benefit and protection against progressive retinal degeneration.

Medical Research: What recommendations do you have for future research as a result of this study?

Prof. Bainbridge : We recognise the obvious potential for gene therapy to treat Leber Congenital Amaurosis and have now developed a new, more powerful gene therapy vector and are aiming to test this in a second LCA2 clinical trial funded by The UK Medical Research Council.

Citation

Bainbridge, JWB, Mehat MS, Sundaram V, et al. Long-term Effect of Gene Therapy on Leber Congenital Amaurosis. Will be published in New England Journal of Medicine. 2015; [Epub ahead of print – available as of May 4, 2015].

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MedicalResearch.com Interview with: Professor James Bainbridge, MA, PhD, FRCOphth (2015). More Powerful Gene Vector Aims To Restore Vision In Inherited Blindness 

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