A key hurdle to making adeno-associated virus (AAV) capsid mediated gene therapy broadly beneficial to all patients is overcoming pre-existing and therapy-induced immune responses to ...

Adeno-associated virus (AAV) capsids have shown clinical promise as delivery vectors for gene therapy. However, the high prevalence of pre-existing immunity against natural capsids poses ...

Modern experimental technologies can assay large numbers of biological sequences, but engineered protein libraries rarely exceed the sequence diversity of natural protein families. Machine learning ...

Efficient design of biological sequences will have a great impact across many industrial and healthcare domains. However, discovering improved sequences requires solving a difficult optimization ...

Machine learning methods are increasingly employed to address challenges faced by biologists. One area that will greatly benefit from this cross-pollination is the problem of ...

Multiplexed barcoded capsid libraries have emerged as a powerful tool for high-throughput AAV engineering. Barcoded studies facilitate the accurate measurement of tropism across multiple capsids ...

Adeno-associated virus (AAV) capsids can deliver transformative gene therapies, but our understanding of AAV biology remains incomplete. We generated the complete first-order AAV2 capsid fitness ...

Advances in DNA delivery will be crucial to the enablement of new gene therapies. Pioneering efforts in developing recombinant AdenoAssociated Virus (AAV) technology for gene ...