Gene Therapy: SUMO to the Rescue

Revolutionizing rAAV production through advanced SUMO fusion technology

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Recombinant adeno associated viruses (rAAV) have become the vehicle of choice to deliver gene therapy for cure genetically inherited diseases but also gene delivery system for many chronic diseases. There are major hurdles in production of safe rAAV.

Generally, rAAV is produced by transfecting three plasmid systems: (1) an AAV2 ITR-containing plasmid carrying the gene of interest; (2), a plasmid that carries the AAV2 Rep-Cap proteins; and (3), a plasmid that provides the helper genes isolated from adenovirus.

The Challenge

This method of producing a therapeutic dose of rAAV is very labor intensive and expensive due to low yield of the rAAV. While progress is being made to manufacture large titers of therapeutic viruses, it is hampered by lack of foreign gene expression or degradation of the targeted protein.

If we can construct a highly potent rAAV that expresses the therapeutic gene, it will solve the problem of large manufacturing batches, decreasing the cost of goods. We believe application of SUMO (Small Ubiquitin-like Modifiers) fusion with therapeutic gene will solve many of the problems described above.

SUMO (Small Ubiquitin-like Modifier) is a member of the ubiquitin family, composed of a flexible N-terminal region followed by a ball-shaped ubiquitin-like fold.

Many therapeutic programs are abandoned due to lack of adequate and cost-effective manufacturing of rAAV. Several factors contribute to dramatically enhance protein production and improve quality of gene delivery.

Nature designed SUMO to chaperone proteins in the cell. Thus, SUMO fusion enables the protein to be stable for a long time to have its biological function. This property allows small doses of rAAV injection to achieve greater therapeutic response.

LifeSensors can guide scientists about the most suitable human SUMO tag and the SUMO protease to efficiently remove SUMO to reduce cost of goods. LifeSensors can guide you from your cloning strategy for the best expression and establish a non-GMP process from gene to rAAV production. The process can be easily adapted for downstream GMP production of the rAAV.

Enhanced Expression

Under expressed genes are efficiently translated

Protein Stability

The gene product is stable at -20C and -80C for several years

Potent rAAV

Development of potent rAAV gene reduces the amount of rAAV required for therapeutic benefit

SUMO Vaccine Presentation

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To use, simply utilize the drop-down arrows in order to search for your target of interest or application category. LifeSensors SUMO technology has been utilized for the expression of many different targets and continues to be utilized worldwide.