Structural, functional, and immunogenicity implications of F9 gene recoding.

ABSTRACT: Hemophilia B is a blood clotting disorder caused by deficient activity of coagulation factor IX (FIX). Multiple recombinant FIX proteins are currently approved to treat hemophilia B, and several gene therapy products are currently being developed. Codon optimization is a frequently used technique in the pharmaceutical industry to improve recombinant protein expression by recoding a coding sequence using multiple synonymous codon substitutions. The underlying assumption of this gene recoding is that synonymous substitutions do not alter protein characteristics because the primary sequence of the protein remains unchanged. However, a critical body of evidence shows that synonymous variants can affect cotranslational folding and protein function. Gene recoding could potentially alter the stru

SUBMITTER: Katneni UK 

PROVIDER: S-EPMC9278298 | biostudies-literature | 2022 Jul

REPOSITORIES: biostudies-literature