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Removal of Empty Capsids from High-Dose Adeno-Associated Virus 9 Gene Therapies

2024-04-14

Abstract excerpt

Recombinant adeno-associated virus, serotype 9 (rAAV9) has shown promise as a gene therapy vector for muscle and central nervous diseases. High-dose requirements of these therapies present critical safety considerations and biomanufacturing challenges. Notably, reduction of empty capsids (ECs), which lack therapeutic transgene, from rAAV9 products is critical to maximize efficacy. Removal of rAAV ECs from full cap...

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Literature Corpus work
535695c3-85e1-50ee-8298-b9ee37cd366c
DOI
10.22541/au.171311478.84577099/v1
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Removal of Empty Capsids from High-Dose Adeno-Associated Virus 9 Gene TherapiesDOI 10.22541/au.171311478.84577099/v1
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