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Expanding Patient Access to Gene Therapies Through AAV Capsid Engineering

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"Expanding Patient Access to Gene Therapies Through AAV Capsid Engineering"

Adeno-associated viral (AAV) vectors present a host of advantages for use as delivery vehicles for genetic material. They also have limitations that impact their safety, efficacy, and applicability.

Capsid engineering can increase AAV capsids' specificity, transduction efficiency, and manufacturability while reducing unwanted immune responses. Such improvements have the potential to broaden the range of diseases that can be treated with gene therapies and greatly expand access to patients.

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