The curious challenges of gene therapy development
In software-speak a bug is a glitch and a feature is an intended functionality.
In gene therapy, however, the ‘bug’ is the virus or – more accurately – the viral vector itself, and the feature or intended functionality is a gene product that the vector carries and/or expresses.
Viral vectors are by far the most prevalent gene carriers owing to superior tissue targeting and transduction efficiency compared to non-viral vectors.
Non-viral vectors, however, like the lipid nanoparticle-based mRNA vaccines for COVID-19 – BNT162b2 (BioNTech-Pfizer) and mRNA-1273 (Moderna) – are also increasingly used because of their lower immunogenicity, easy preparation and low cost.
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