Patent ID: 7517644
Filing Date: 2009-04-14
Classification: A61P,C12N,C12Q

Abstract:
1. A method for rational discovery of antisense oligonucleotides useful as therapeutics for the treatment of an Aberrant Programming Disease wherein said Aberrant Programming Disease is cancer, comprising the steps of (i) selecting one or more transcriptional regulator gene targets implicated in the regulation of cellular programming, said transcriptional regulator targets are not oncogenes and are expressed by cancer cells to be reprogrammed; (ii) selecting one or more prototype antisense oligonucleotides(s) to target transcripts of the selected non-oncogene transcriptional regulators, where said prototype antisense oligonucleotide(s) is selected from the group of oligonucleotides comprising SEQ ID NO: 1 through SEQ ID NO: 3626; (iii) evaluating said prototype antisense oligonucleotide(s) in a Reprogramming Test, alone and then in combination with an augmentation agent, for capacity to therapeutically reprogram the Aberrant Programmed disease cells while not adversely reprogramming normal cells, said augmentation agent being capable of altering the pattern of transcriptional regulator expression in the Aberrant Programmed disease cells; (iv) selecting sequence- and length-variants of prototype oligonucleotides scored in the Reprogramming Test to exhibit capacity to reprogram Aberrant Programming cells; (v) evaluating said variants for efficacy in the Reprogramming Test, either alone or in combination with the appropriate augmentation agent; (vi) selecting the most active of the evaluated antisense oligonucleotides, or antisense oligonucleotide/augmentation agent combination, and testing the same in vivo for efficacy.