Journal
EXPERT OPINION ON ORPHAN DRUGS
Volume 3, Issue 7, Pages 787-798Publisher
TAYLOR & FRANCIS LTD
DOI: 10.1517/21678707.2015.1046434
Keywords
choroideremia; gene therapy; REP1; retina
Categories
Funding
- NIHR Biomedical Research Centre (Oxford and Moorfields)
- Wellcome Trust
- UK Department of Health
- CIHR Emerging Team
- Choroideremia Research Foundation Canada Inc.
- Alberta Innovates - Health Solutions Collaborative Research and Innovation Opportunities (CRIO) Project
- FFB Canada
- Alberta Innovates [201201139] Funding Source: researchfish
- National Institute for Health Research [12/66/35] Funding Source: researchfish
Ask authors/readers for more resources
Introduction: Choroideremia is a rare, X-linked disorder recognized by its specific ocular phenotype as a progressive degenerative retinopathy resulting in blindness. New therapeutic approaches, primarily based on genetic mechanisms, have emerged that aim to prevent the progressive vision loss. Areas covered: This article will review the research that has progressed incrementally over the past two decades from mapping to gene discovery, uncovering the presumed mechanisms triggering the retinopathy to preclinical testing of potential therapies. Expert opinion: While still in an evaluative phase, the introduction of gene replacement as a potential therapy has been greeted with great enthusiasm by patients, advocacy groups and the medical community.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available