Potential lifetime quality of life benefits of choroideremia gene therapy: projections from a clinically informed decision model.

Celine-Lea Halioua-HauboldJasleen Kaur Jolly James Andrew Smith Rafael Pinedo-VillanuevaDavid A BrindleyRobert E MacLaren

Published in: Eye (London, England) (2019)

In young adults in the earlier stages of choroideremia, successful gene therapy is expected to provide a significant increase in health-related quality of life.

Keyphrases

gene therapy
young adults
decision making
human health
risk assessment
climate change