Key result
Viral vectors, particularly recombinant adeno-associated viruses and adenoviral vectors, are emerging as promising delivery vehicles for programmable nucleases to correct Duchenne muscular dystrophy.
Viral vectors show promise for delivering gene-editing tools like CRISPR-Cas9 to correct DMD mutations in vivo and ex vivo, but immunological and delivery hurdles must be overcome for clinical translation.
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May advance AAV-based gene editing for DMD; leaves open clinical translation due to immunogenicity and delivery barriers.
Maggio et al. (2016) conducted a review in Duchenne muscular dystrophy (DMD). Viral vectors for gene editing (AdVs, rAAVs) was evaluated. Viral vectors, particularly recombinant adeno-associated viruses and adenoviral vectors, are emerging as promising delivery vehicles for programmable nucleases to correct Duchenne muscular dystrophy.
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