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AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice - Nature

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AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice - Nature

Gene therapy is a potentially curative medicine for many currently untreatable diseases, and recombinant adeno-associated virus (rAAV) is the most successful gene delivery vehicle for in vivo applications1–3. However, rAAV-based gene therapy suffers from several limitations, such as constrained DNA cargo size and toxicities caused by non-physiological expression of a transgene4–6.

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AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice - Nature

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AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice - Nature

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