Gene Therapy is experiencing a resurgence in both the research and pharmaceutical communities. Just in the last few weeks, gene therapy for five infants with severe combined immunodeficiency has been reported as a success and two new gene therapy start-up companies have been launched. This sea-change has been powered by elegant studies in which new viral delivery systems – such as lentiviral vectors containing cell-fate control safety cassettes -- deliver potent payloads by using codon optimization to improve the expression levels of therapeutic genes in human cells.


Historically, gene therapies have been plagued by several challenges. The emergence of leukemia in former gene therapy patients has been attributed to disruption of oncogenes when etroviral vectors randomly inserted the therapeutic genes into the genome. On the other hand, gene therapies have often failed to reach the desired levels of efficacy, either because gene delivery is disrupted when viral vectors are detected and disarmed by the immune system or because the delivered genes are expressed at levels too low to prove therapeutic.

Deliver genes optimized for expression with OptimumGene from GenScript


Researchers have chosen GenScript's patented OptimumGene™ codon optimization algorithm to generate therapeutic RAG2 transgenes for SCID, secreted antiviral proteins that inhibit HIV infection, α-galactosidase A expression to correct Fabry Disease, and many other gene therapies.