Recombinant adeno-associated virus (rAAV) vectors for delivering therapeutic genes have demonstrated their safety in multiple diseases and clinical settings over the years and are a proven and effective tool that can be used to deliver new gene editing and replacement and genome modification technologies. The combination of more tailored rAAV delivery vectors and new gene knockdown and editing techniques will enable unique approaches to the therapeutic manipulation of gene expression, as described in an article in Human Gene Therapy.
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