It's what's on the inside that counts: Capsid content as a critical quality attribute for AAV characterization
Lauriel F Earley1, Stuart Nelson2, Ana Dolinar Češarek3
1Earley Stage Gene Therapy Consulting, Tacoma, WA, USA.
Summary
Recombinant adeno-associated virus (rAAV) vector characterization needs better methods to assess genome integrity. Current assays are insufficient, impacting gene therapy safety and efficacy.
Area of Science:
- Biotechnology
- Molecular Biology
- Gene Therapy
Background:
- Recombinant adeno-associated virus (rAAV) vectors are crucial for gene therapy but exhibit heterogeneity.
- Existing analytical methods for rAAV capsid attributes are diverse but lack comprehensive assessment.
- Current strategies often neglect genome integrity, a critical quality attribute.
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