High-throughput 5' UTR engineering for enhanced protein production in non-viral gene therapies

Nat Commun. 2021 Jul 6;12(1):4138. doi: 10.1038/s41467-021-24436-7.

Abstract

Despite significant clinical progress in cell and gene therapies, maximizing protein expression in order to enhance potency remains a major technical challenge. Here, we develop a high-throughput strategy to design, screen, and optimize 5' UTRs that enhance protein expression from a strong human cytomegalovirus (CMV) promoter. We first identify naturally occurring 5' UTRs with high translation efficiencies and use this information with in silico genetic algorithms to generate synthetic 5' UTRs. A total of ~12,000 5' UTRs are then screened using a recombinase-mediated integration strategy that greatly enhances the sensitivity of high-throughput screens by eliminating copy number and position effects that limit lentiviral approaches. Using this approach, we identify three synthetic 5' UTRs that outperform commonly used non-viral gene therapy plasmids in expressing protein payloads. In summary, we demonstrate that high-throughput screening of 5' UTR libraries with recombinase-mediated integration can identify genetic elements that enhance protein expression, which should have numerous applications for engineered cell and gene therapies.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • 5' Untranslated Regions / genetics*
  • Algorithms
  • Cell Line
  • Gene Expression
  • Genetic Engineering*
  • Genetic Therapy*
  • HEK293 Cells
  • High-Throughput Screening Assays
  • Humans
  • Plasmids
  • Promoter Regions, Genetic
  • Recombinases

Substances

  • 5' Untranslated Regions
  • Recombinases

Associated data

  • figshare/10.6084/m9.figshare.14624472.v1