AAVM41 Serotype

Recombinant AAV capsid of AAV1, 6, 7 and 8 origin, selected from a shuffled AAV1-9 capsid library by in vivo panning in mice for striated muscle targeting. After systemic delivery, myocardium shows the highest expression among tissues tested.

Length: 1 bp

Tropism: Myocardium (highest expression), heart and skeletal muscle; non-muscle tissues, mainly liver, dramatically reduced

Characteristics

Capsid M41 was retrieved after 2 rounds of in vivo selection by tail vein injection in mice, based on high frequency in muscle and low in liver. It has a mosaic capsid surface and a conserved interior. After i.v. reporter delivery it is more efficient than AAV6 in heart and muscle, similar to AAV9 in heart, weaker than AAV9 in muscle, with dramatically reduced non-muscle (mainly liver) transfer.

Applications: Systemic gene delivery to myocardium in mice and hamsters. In a genetic cardiomyopathy hamster model, AAVM41 achieved efficient long-term delta-sarcoglycan gene expression and rescue of cardiac functions. Selection by in vivo panning of capsid libraries was proposed for de-targeting and retargeting vector tissue tropism.

Limitations: Weaker than AAV9 in skeletal muscle after i.v. delivery in mice, with myocardium as the highest-expressing tissue, so it suits cardiac targeting better than skeletal muscle targeting. The abstract compares it only with AAV6 and AAV9.

Related collections

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