CanScript for Gene Therapy: a DNA Enhancer Element Conveying Cancer-specific Expression to 30% of Human Malignancies
One of the major requirements for effective cancer gene therapies are DNA sequences that, when inserted into a gene-expression construct, will drive high-level gene expression exclusively in cancer cells. These sequences are termed cancer specific enhancers or promoters of gene transcription. Until now, no highly specific enhancer has been identified that can drive high levels of gene expression in greater than 10% of human malignancies. An invention by JHU scientists now describes a short DNA sequence - CanScript, a cancer-specific enhancer of mRNA expression that drives expression in over 30% of human malignancies. The enhancer is modular, meaning it can be conveyed to other vectors to drive high-level expression of desired genes in a cancer-specific manner. A single copy of this DNA sequence elevates gene expression about 10-fold in cancer cells expressing the cancer-specific gene. It is expected that a concatemer of CanScript would have far greater activity. The enhancer element identified will be useful for gene therapy of many human cancers, including the vast majority of pancreatic cancers, most serous ovarian cancers, nearly half of lung cancers, about 30% of colorectal cancers, and 15% of breast cancers. Description (Set) Proposed Use (Set) CanScript DNA sequence enables a gene-therapy vector to achieve high-level cancer-specific expression of a therapeutic gene, and may also be used to augment replication-competence in replicative infectious vectors. CanScript may also be used for diagnostic purposes, as a vector may include the CanScript DNA sequence to drive the expression of a gene useful in diagnostic imaging. In addition, a monitoring vector may include the CanScript DNA sequence to report the activity of the cancer-specific protein, especially if one were attempting therapeutically to modify the signaling pathway governing the protein�s expression.Inventor(s): Kern, Scott
Type of Offer: Licensing
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