Mir‐142‐3p target sequences reduce transgene‐directed immunogenicity following intramuscular adeno‐associated virus 1 vector‐mediated gene delivery

Muscle represents an important tissue target for adeno‐associated virus (AAV) vector‐mediated gene transfer in muscular, metabolic or blood‐related genetic disorders. However, several studies have demonstrated the appearance of immune responses against the transgene product after intramuscular AAV vector delivery that resulted in a limited efficacy of the treatment. Use of microRNAs that are specifically expressed in antigen‐presenting cells (APCs) is a promising approach for avoiding those immune responses. Cellular mir‐142‐3p, which is APC‐specific, is able to repress the translation of its target cellular transcripts by binding to a specific target sequences.

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