Insulation Cassette that Enables Controlled, Sustained Gene Therapy Delivery

Cell-Type Specific Therapy Targets Cells that Need Therapy and Leaves Non-Target Cells Unaffected

This gene delivery cassette allows for the controlled and sustained expression of a gene. The field of gene therapy, which tries to use engineered viruses to defeat disease at the DNA level, offers a promising solution for the treatment of a wide spectrum of human diseases. Interest from venture capital firms is growing; in 2014, U.S. companies raised a total of $600 million. The gene therapy market is likely to be worth $11 billion by 2025. University of Florida researchers have engineered a gene expression cassette that can insulate the therapeutic genetic material inside from the influence of surrounding genetic material. The cassette is also highly controllable and can be targeted to specific cell types, representing a major technological step and a promising business opportunity in the field of gene therapy.

Application

Insulation cassette for sustained and regulatable gene delivery

Advantages

• Permits maintenance of persistent, highly controllable, long-term gene expression, significantly improving quality and effectiveness of gene therapy products
• Enables cell-type specificity, assuring therapeutic resources are used directly on the cells needing them, while leaving non-target cells unaffected
• Vector system packages large DNA insertions, efficiently providing the needed space for complex genetic information
• Can be used for the production of large amounts of a needed gene for pharmacological or agricultural purposes in animals or plants
• Can be engineered to combat non-central nervous system disorders, offering opportunitiesin a broad range of other markets

Technology

This gene therapy delivery cassette employs a defective form of a virus as the vector to carry the gene expression cassette for gene transfer to the central and peripheral nervous systems. Specifically, the technology allows a transgene to maintain persistent, long-term and highly regulatable gene expression, unhindered by host cell silencing mechanisms such as histone methylation/deacetylation, DNA methylation, position effects, or transgene copy number. The delivery system is thus composed of (1) the insulated gene expression cassette and (2) a defective virus-based virus vector for delivery of the transgene to the central nervous system. The insulation cassette has the potential to become a powerful tool in the field of gene therapy, basic gene expression assays, and development of animal disease models. Note that the insulated gene cassette can be used alone, such as in transgenic applications, or be used with other DNA-based or viral delivery systems. Related to 11434