Gene Promoter allows Viral Vectors to Transmit Double-Stranded DNA

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Researchers
William W. Hauswirth
Sanford L. Boye
Barry J. Byrne
Managed By
April Kilburn
Assistant Director 352-392-8929
Patent Protection
US Patent 8,298,818

Reduces Time for Cells to Express the Gene Product

Invention

This gene promoter can be combined with self-complementary adeno-associated viruses (scAAVs) to transmit double-stranded DNA to target cells. Gene therapy is one of the quickest moving fields in science with an annual growth rate of 48.9%, and is expected to reach a market value of $11 billion by 2025. The rate limiting step for delivery of genes from a viral vector to the creation of a gene product is conversion by the host cell of virally transmitted single stranded DNA to double-stranded DNA which is usable by the host. Researchers at the University of Florida have developed a small, ubiquitous gene promoter that when combined with scAAVs solves this problem. Because the viral vector transmits double-stranded DNA, there is a great reduction in the time it takes the cell to express the gene product.

Applications

Gene promoter increases speed of gene expression by transmitting double-stranded DNA to cells

Advantages

  • Virus is pre-packaged with double stranded DNA, thus greatly reducing the time necessary to express the gene product.
  • Recombinant AAV (rAAV) is used as the vehicle for delivery of genetic materials, therefore it has no associated pathological human condition and does not contain native viral coding sequences resulting in persistent expression of delivered transgenes.
  • Has the ability to be used with scAAV which allows for expression to be initiated much sooner than with standard AAV and with greater efficiency;
  • Expresses in a wide variety of cells which, when used in conjunction with different AAV serotypes results in the ability to target a broad range of tissues;
  • Has the capacity to promote expression for long periods post infection which reduces the necessity for re-infection

Technology

Current methods for gene delivery are greatly inefficient. The rate limiting step for introduction of a gene into cells is the conversion from single stranded DNA to double stranded DNA. Researchers at the University of Florida have solved this problem by creating a long acting, small, ubiquitous promoter that has the ability to be packaged into adeno-associated viruses in double stranded form. This promoter has the ability to initiate gene expression efficiently while keeping expression levels high for a long duration Novel Gene Delivery Method of time, and also allow investigators to transduce a wide variety of cell types. Because this promoter is packaged into AAV, there is no known pathological condition that is known to be associated with vector transfection. In summary, this promoter is not associated with common human pathologies, and has the ability to express efficiently and quickly while maintaining a long expression period.