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EXPANDING PATIENT ACCESS TO GENE THERAPIES THROUGH AAV CAPSID ENGINEERING

EXPANDING PATIENT ACCESS TO GENE THERAPIES THROUGH AAV CAPSID ENGINEERING

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Adeno-associated viral (AAV) vectors are crucial in gene therapy, but natural AAV capsids have limitations, including poor tissue specificity and immune response issues. Capsid engineering aims to enhance these vectors by improving specificity, transduction efficiency, and manufacturability while reducing immune reactions. These advancements can broaden the applicability of gene therapies, allowing more patients to benefit. Companies like Porton Advanced Solutions are leading efforts in developing engineered capsids with superior performance. Their work includes creating novel capsids that evade preexisting antibodies, target specific tissues more effectively, and enhance gene therapy's overall safety and efficacy.

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