AUG 21, 2013 01:00 PM PDT

Codon optimization: new safety concerns for gene therapy and genetic vaccines

  • Associate Professor of Neurobiology, Scripps Institute
      Dr. Mauro is an Associate Professor in the Department of Neurobiology at the Scripps Research Institute in La Jolla, California. He is also a co-founder and lead scientist of Promosome, a biotechnology company focused on bioproduction enablement and DNA vaccines. In addition, Dr. Mauro is a Senior Fellow in Experimental Neurobiology at the Neurosciences Institute in San Diego, California. Prior to moving to The Scripps Research Institute, Dr. Mauro received his Ph.D. at McGill University in Montreal, Quebec, and continued his studies as a postdoctoral fellow at The Rockefeller University in New York City. Dr. Mauro studies both fundamental and applied aspects of translational control mechanisms. His basic research is focused on understanding how eukaryotic mRNAs recruit ribosomes, how ribosomes subsequently locate initiation codons, and how ribosomes regulate the translation of specific subsets of mRNAs. Dr. Mauro's applied studies build on his basic research. These applied studies have led to the identification of Translational Enhancer Elements (TEEs) and the generation of synthetic translational enhancers.


    Codon-optimization describes gene engineering approaches that use synonymous codon changes to increase protein production. It is used extensively for expressing recombinant protein drugs, gene therapy, and DNA/RNA vaccines. However, it is becoming increasingly clear that codon-optimization can affect protein conformation and function, increase the immunogenicity of recombinant protein drugs, and reduce efficacy. This presentation will analyze the mechanisms and assumptions underlying codon optimization and identify additional hazards associated with this approach, including unique hazards for gene therapy and DNA/RNA vaccine applications.

    Show Resources
    Loading Comments...