A No-Nonsense Approach to Inherited Disease
A new generation of non-viral vectors can help treat more genetic disorders while making gene therapy easier and safer – and new treatment modalities promise to entirely change the management of inherited eye disease
Mariya Moosajee | | Hot Topic
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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