Morpholino Antisense Induced Translational Frameshifting

Premature stop codon and framshift mutations account for approximately 25% of all cases of genetic disease. Unfortunately, therapeutic approaches to diseases caused by frameshift and nonsense mutations have been very limited and difficult to implement; as such, there is a need for a method of treating such mutations/diseases. This invention is designed to suppress the phenotypic effects of such mutations by manipulating the process of protein synthesis. Exposing an mRNA to morpholino antisense oligonucleotides can result in ribosomal frame shift. A ribosomal frameshift occurring near a frameshift mutation returns the ribosome to the correct reading frame and results in a production of full length protein. If the full length protein were produced in sufficient quantities, it would ameliorate the disease symptoms.

There is a growing market for DNA/RNA technology that is predicted to hit $1.2 billion by 2010. Two of the main categories are microarrays and antisense technology. Antisense drugs have the potential to treat a number of disorders that have immense social and economic impacts on the world.

Stage of Development
A formal patent application has been filed.
This technology is part of an active and ongoing research program and has been demonstrated to work in proof-of-concept experiments which include a working prototype. It is available for developmental research support or licensing under either exclusive or non-exclusive terms.

Additional Info
*Howard MT, Gesteland RF, Atkins JF. (2004) Efficient stimulation of site-specific ribosome frameshifting by antisense oligonucleotides. RNA; *

Inventor(s): John Atkins, Michael Howard

Type of Offer: Licensing

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