Cell & Gene Therapy 2021

How is Capillary Electrophoresis (CE) Being Used in Gene Therapy?


( Melovic1
1 Ramcon, Sweden


Over the last 24 months the number of applications of CE in
gene therapy has increased rapidly. Initially being used to
profile not only size but also shape of DNA and RNA oligo
product, it is now being developed for intact viral product
profiling and viral particle analysis. In Figures 1A and 1B are
examples where CE has been used in the purity profiling of
mRNA needed for CRIPSR (1A) and how it has been used
in the analysis of DNA sample (1B) and highlights how it can
be used to show the base or base pair number present in
these samples. Plasmids, a form of oligonucleotides, are
also used in AAV particle manufacturing and CE has been
used for purity profiling in this area by several companies.
Figure 2 highlights the reproducibility of this approach used
to calculate the % of supercoiled version in the final products
with clear separation of other forms including linear or open
circular observed. Finally CE is being used to analyze the
AAV particle itself using CE-SDS profiling of the viral
proteins (Figure 3A) which make up the particle. More
recently it is being developed to separate empty vs. full
capsids (Figure 3B) which is important attribute which
currently requires lengthy and more manual approaches for
example analytical centrifugation to provide data for this

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