Adjusting Plasmid Ratios to Increase Gene Therapy Vector Yield – UROP Symposium

Adjusting Plasmid Ratios to Increase Gene Therapy Vector Yield

Sandra Alcini

Research Mentor: Anna Maurer
Mentor Department: Biophysics, LSA
Author(s): Sandra Alcini, Anna C Maurer
Session: Session 4 (1:00 PM – 1:50 PM)
Presentation Type: Poster 4

Abstract

An Adeno-Associated Viral (AAV) vector is a delivery method for genetic material into a patient’s body. To generate AAV vectors, three plasmids are introduced into the cell in a 2:1:1 ratio: one encoding required Adenoviral factors, one encoding the AAV viral genes, and one encoding the delivery gene of interest. When expressed in a cell, these genes produce a recombinant AAV vector, packaged with a gene of interest. The rAAV vector is then deliverable to the patient by therapeutic dose. Doses are, however, still very expensive to produce. This study tests different plasmid ratios to observe whether changing the ratios will yield higher vector titer (vector genomes per milliliter, Vg/mL), thereby lowering the cost of production and making AAV gene therapy a more attainable option.

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