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Prediction of Adeno-Associated Virus Fitness with a Protein Language-Based Machine Learning Model.

Human gene therapy - 1 May 2025

Wu Jason, Qiu Yu, Lyashenko Eugenia, Torregrosa Tess, Pfister Edith L, Ryan Michael J, Mueller Christian, Choudhury Sourav R

Abstract excerpt

Adeno-associated virus (AAV)-based therapeutics have the potential to transform the lives of patients by delivering one-time treatments for a variety of diseases. However, a critical challenge to their widespread adoption and distribution is the high cost of goods. Reducing manufacturing costs by developing AAV capsids with improved yield, or fitness, is key to making gene therapies more affordable. AAV fitness...

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PMID: 40241334
DOI: 10.1089/hum.2024.227

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