Some genes are too large to fit into AAV vectors. UCB, Novartis back $57M idea to deliver them in parts instead

Amber Tong

Senior Editor

Of all the major limitations of adeno-associated viruses (AAV) as a vector for gene therapy, one is particularly well-known and indisputable: They can only carry a transgene up to a certain size. Design a payload larger than 4.7kb, and there’s simply no way you can fit everything in.

That’s why gene therapy developers working on, say, Duchenne muscular dystrophy have come up with workarounds, such as creating shortened versions of the target gene.

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Some genes are too large to fit into AAV vectors. UCB, Novartis back $57M idea to deliver them in parts instead

Amber Tong

Senior Editor

Unlock this article instantly by becoming a free subscriber.

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Some genes are too large to fit in­to AAV vec­tors. UCB, No­var­tis back $57M idea to de­liv­er them in parts in­stead

Amber Tong

Senior Editor

Unlock this article instantly by becoming a free subscriber.

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