A hop, skip, and a jump to evade USH2A deaf-blindness mutations

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Over the past decade, many gene therapy approaches have been designed to restore auditory and visual function lost due to Usher syndrome (USH).1 Among them, gene replacement therapy mediated by adeno-associated virus (AAV)-based vectors has been shown to be a safe and efficient strategy. However, this approach is not suitable for large genes, such as USH2A, which vastly exceed the cargo capacity of AAV vectors. In this issue of Molecular Therapy, Dulla et al.2 provide evidence that synthetic antisense oligonucleotides (ASO or AON) can mediate exon-skipping, which may be a viable approach for a subset of USH2A mutations.