Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
AssigneeEtagen Pharma, Inc.
InventorsTod M. Woolf, Alexandre V. Lebedev, Richard I. Hogrefe
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AssigneeEtagen Pharma, Inc.
InventorsTod M. Woolf, Alexandre V. Lebedev, Richard I. Hogrefe
Estimating the term…
The present invention includes compositions and methods for the treatment of a medical condition or disease utilizing editing oligonucleotides. The editing oligonucleotides contain a an oligonucleotide strand of about 10 to about 50 nucleotides on each side of the editing moiety which may contain a sugar or linker that positions the active editing moiety in the proper location for hybridization to the target nucleic acid. The editing oligonucleotides may also contain at least one nucleotide sequence change from the targeted sequence in the genome. The method includes modifying a genomic sequence within a cell utilizing an editing oligonucleotide without additional proteins or nucleic acids to assist in the editing process. The editing oligonucleotide may comprise backbone modifications that increase the nuclease stability of the oligonucleotide as compared to unmodified oligonucleotides or oligonucleotides having three phosphorothioates on each terminus.
A method of modifying a genomic sequence within a cell or an individual comprising the steps of:
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A method of improving the health of an individual requiring treatment for a medical condition, for reducing or eliminating a medical condition in an individual requiring treatment for said condition, for reverting a mutated nucleotide in a target nucleic acid to the wild-type nucleotide, for modifying a non-mutated nucleotide of a mutated codon in a target nucleic acid to produce a wild-type codon, for converting a stop codon in a target nucleic acid to a read through non-wild type codon, for modifying a mutated codon in a target nucleic acid to produce a non-wild type codon that results in a non-disease causing amino acid, for modifying a nucleic acid encoding a protein to modulate said protein's activity, for modifying a nucleic acid encoding a mutant protein to suppress its disease causing affects comprising:
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An editing oligonucleotide for site-specific modification of a target nucleic acid comprising the structure:
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Dependencies are inferred from the claim text.