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Biotech / Medical : Gene therapy
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To: Mike McFarland who wrote ()5/11/2000 4:49:00 AM
From: nigel bates  Read Replies (1) of 319
 
Came across this (60% owned subsidiary of UK based Proteome Sciences).

intronn.com
Intronn has developed and patented a new technology for gene therapy that exploits the differential gene expression between normal and diseased cells. It is called Spliceosome Mediated RNA Trans-splicing Gene Therapy or SMaRT Gene Therapy. This technology may be applied to a wide range of diseases that involve the expression of unique or mutated genes. By using SMaRT Gene Therapy, virtually any gene (pre-mRNA) may be targeted and reprogrammed to produce a new gene product useful in treating that disease. SMaRT technology can be employed to control the expression of any delivered gene by the presence of the chosen target pre-mRNA.
In the case of cancer or other conditions, such as HIV infection, where removal (death) of the diseased cells would be beneficial, the target gene can be reprogramed to encode a therapeutic gene product that kills the diseased cells. For genetic diseases, Intronn can repair the defective genes, allowing target cells to make the correct proteins. Intronn scientists have already achieved gene (mRNA) repair in cultured human cells using a lac Z (reporter gene) model system. We are currently applying SMaRT repair to the genetic defects responsible for a prevalent lethal genetic disease.
Intronn LLC has developed pre-therapeutic RNA molecules (PTMs) that are capable of inducing SMaRT reactions with specific target pre-mRNAs. We have produced a series of PTMs capable of base-pairing to and trans-splicing with disease associated pre-mRNA targets. Using SMaRT Gene Therapy, Intronn scientists have successfully reprogramed a cancer associated gene to encode diphtheria toxin, resulting in the death of the cancer cells. These PTMs have caused SMaRT reactions in human cell nuclear extract (in vitro), in cultured H1299 human lung cancer cells and in H1299 tumor bearing athymic (nude) mice. RT-PCR results and sequence analysis of the trans-spliced products demonstrated that accurate trans-splicing occurred between the PTMs and the cancer associated pre-mRNA target both in vitro and in vivo. The trans-spliced mRNA has been shown to encode the first exon of the cancer gene linked to the entire coding sequence of diphtheria toxin. Upon translation into protein, such mRNAs are lethal to cancer cells.
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