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Subspecialties Retina, Health Economics and Policy, Business and Innovation, Basic & Translational Research

A No-Nonsense Approach to Inherited Disease

At a Glance

  • Gene therapy for inherited eye diseases of the eye is conceptually attractive, but has drawbacks relating to vector characteristics and health economics
  • Now, innovative vectors both circumvent the transgene silencing issue associated with legacy non-viral vectors and avoid the capacity limitations, immunogenicity and insertional mutagenesis potential of viral vectors
  • At the same time, new classes of therapeutics – including nonsense suppressors – promise the ability to ameliorate multiple inherited diseases with a single drug
  • Together, these advances are likely to provide patients with new and effective options and to transform both the management of genetic disease and the economics of the gene therapy sector.

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About the Author

Mariya Moosajee

Mariya Moosajee is a Consultant Ophthalmologist and Associate Professor at Moorfields Eye Hospital, Great Ormond Street Hospital for Children and UCL Institute of Ophthalmology, London, UK.

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