Diversifying gene therapy vectors with machine learning
The capsid of Adeno-associated Virus (AAV) is a naturally occurring, replication-deficient, virus that is widely considered the frontrunner for solving the delivery problem in gene therapy.
The capsid of Adeno-associated Virus (AAV) is a naturally occurring, replication-deficient, virus that is widely considered the frontrunner for solving the delivery problem in gene therapy. These viruses are known to be harmless to humans, and are relatively simple to manipulate. One well-known drawback of natural capsids however, which are currently used for delivery, is that many patients with pre-existing immunity to the virus (due to previous natural exposure) may be ineligible for life-changing treatment. In previous work (published in Science), we validated the use of computational…
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