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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.
As many inherited diseases are very rare, the development costs are spread over smaller numbers of patients – which translates into very high therapy prices.
I believe that our non-viral, S/MAR-containing vectors should be safer, more effective and more broadly applicable than historical gene therapy vectors.
In some diseases – for example, aniridia – 40 percent of patients exhibit premature stop codons in the causative gene.

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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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