Kumaran, N;
Smith, AJ;
Michaelides, M;
Ali, R;
Bainbridge, J;
(2018)
Gene therapy for Leber congenital amaurosis.
[Review].
Expert Review of Ophthalmology
, 13
(1)
pp. 11-15.
10.1080/17469899.2018.1429916.
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Abstract
Introduction: Leber congenital amaurosis (LCA) is a group of recessively inherited, early infantile-onset, severe rod-cone dystrophies that can result from defects in at least 25 genes, including RPE65, CEP290, RDH12, AIPL1 and GUCY2D. The possibility of benefit is offered by therapeutic intervention to provide the functional gene that is otherwise lacking. Areas covered: We searched PubMed for publications using the relevant keywords. Expert commentary: Clinical trials of gene therapy for LCA owing to defects in RPE65 have demonstrated benefit with improved function of rod photoreceptor cells. A gene therapy for this condition has been approved by the FDA. Ongoing clinical trials aim to determine whether cone photoreceptor cell function can be protected by appropriate gene delivery at an early stage of the disease. Clinical trials of gene therapy for LCA owing to defects in 5 other genes are planned.
| Type: | Article |
|---|---|
| Title: | Gene therapy for Leber congenital amaurosis |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1080/17469899.2018.1429916 |
| Publisher version: | http://doi.org/10.1080/17469899.2018.1429916 |
| 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: | Leber congenital amaurosis, LCA, LCA2, RPE65 associated LCA, gene therapy, clinical trials |
| 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/10046441 |
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