Oligonucleotides as Tools for Chemotherapy

Massachusetts General Hospital, Boston MA

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Abstract

DESCRIPTION (provided by applicant): Completion of sequencing of the human and of bacterial genomes has made it possible to apply the principle of oligonucleotide hybridization competition to the inhibition of nefarious genic expression. We shall focus our efforts on three targets: l) M. tuberculosis, with its uniquely constructed mycocerosic outer cell wall; 2) Cystic Fibrosis, in which for our novel oligonucleotide insertion technique we must pin down the extent of insertion. Present estimates, as a result of over thousand sequencings, are that insertion occurs in 10-25 percent of delta508 mRNA molecules; 3) Huntington's Disease, requiring further studies to firm up our finding of inhibition of expression of Huntington protein in tissue cultures from patients with this disease, using derivatives of antisense oligonucleotides.