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This research aims to understand the factors limiting AAV capsid production in gene therapy.

AAV Assembled Capsids Are Produced in Cells Blocked From Cell Cycle Progression

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This research aims to understand the factors limiting AAV capsid production in gene therapy.
Characterized cell populations from AAV-transfected HEK293 cells
Compared cell cycle profiles between capsid-producing and non-producing cells
Conducted RNA-seq analysis to identify differences in molecular pathways
Only 10% of transfected HEK293 cells produce assembled AAV capsids
Distinct cell cycle profiles were identified between the two cell populations
RNA-seq analysis highlighted differences in molecular pathways involved in capsid assembly

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Analysis reveals gene therapy production is impacted by cell cycle blockage, suggesting molecular pathway involvement.

Mullick et al. (2025) studied this question.

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