Treatment of interferon-related developmental regulator 1 (ifrd1) related diseases by inhibition of natural antisense transcript to ifrd1
AssigneeCuRNA, Inc.
InventorsJoseph Collard, Olga Khorkova Sherman, Carlos Coito
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Abstract
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Interferon-related developmental regulator 1 (IFRD1), in particular, by targeting natural antisense polynucleotides of Interferon-related developmental regulator 1 (IFRD1). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of IFRD1.
Claims39
A method of modulating a function of and/or the expression of an Interferon-related developmental regulator 1 (IFRD1) polynucleotide in a biological system comprising:
- contacting said system with at least one antisense oligonucleotide 5 to 30 nucleotides in length wherein said at least one oligonucleotide has at least 50% sequence identity to a reverse complement of a natural antisense of an Interferon-related developmental regulator 1 (IFRD1) polynucleotide;
- thereby modulating a function of and/or the expression of the Interferon-related developmental regulator 1 (IFRD1) polynucleotide.
A method of modulating a function of and/or the expression of an Interferon-related developmental regulator 1 (IFRD1) polynucleotide in patient cells or tissues in vivo or in vitro comprising:
- contacting said cells or tissues with at least one antisense oligonucleotide 5 to 30 nucleotides in length wherein said oligonucleotide has at least 50% sequence identity to an antisense oligonucleotide to the Interferon-related developmental regulator 1 (IFRD1) polynucleotide;
- thereby modulating a function of and/or the expression of the Interferon-related developmental regulator 1 (IFRD1) polynucleotide in patient cells or tissues in vivo or in vitro.
A method of modulating a function of and/or the expression of an Interferon-related developmental regulator 1 (IFRD1) polynucleotide in a biological system comprising:
- contacting said system with at least one antisense oligonucleotide that targets a region of a natural antisense oligonucleotide of the Interferon-related developmental regulator 1 (IFRD1) polynucleotide;
- thereby modulating a function of and/or the expression of the IFRD1 polynucleotide.
A method of modulating a function of and/or the expression of an Interferon-related developmental regulator 1 (IFRD1) gene in mammalian cells or tissues in vivo or in vitro comprising:
- contacting said cells or tissues with at least one short interfering RNA (siRNA) oligonucleotide 5 to 30 nucleotides in length, said at least one siRNA oligonucleotide being specific for an antisense polynucleotide of an Interferon-related developmental regulator 1 (IFRD1) polynucleotide, wherein said at least one siRNA oligonucleotide has at least 50% sequence identity to a complementary sequence of at least about five consecutive nucleic acids of the antisense and/or sense nucleic acid molecule of the IFRD1 polynucleotide;
- and, modulating a function of and/or the expression of Interferon-related developmental regulator 1 (IFRD1) in mammalian cells or tissues in vivo or in vitro.
A method of modulating a function of and/or the expression of Interferon-related developmental regulator 1 (IFRD1) in mammalian cells or tissues in vivo or in vitro comprising:
- contacting said cells or tissues with at least one antisense oligonucleotide of about 5 to 30 nucleotides in length specific for noncoding and/or coding sequences of a sense and/or natural antisense strand of an IFRD1 polynucleotide wherein said at least one antisense oligonucleotide has at least 50% sequence identity to at least one nucleic acid sequence set forth as SEQ ID NOS:
- 1 to 7;
- and, modulating the function and/or expression of the IFRD1 in mammalian cells or tissues in vivo or in vitro.
A synthetic, modified oligonucleotide comprising at least one modification wherein the at least one modification is selected from:
- at least one modified sugar moiety;
- at least one modified internucleotide linkage;
- at least one modified nucleotide, and combinations thereof;
- wherein said oligonucleotide is an antisense compound which hybridizes to and modulates the function and/or expression of an Interferon-related developmental regulator 1 (IFRD1) gene in vivo or in vitro as compared to a normal control and wherein said oligonucleotide has at least 50% sequence identity to a sequence of at least about five consecutive nucleic acids that is complementary to the antisense and/or sense nucleic acid molecule of the Interferon-related developmental regulator 1 (IFRD1) polynucleotide and alleles, homologs, isoforms, variants, derivatives, mutants, fragments, or combinations thereof.
A method of preventing or treating a disease associated with at least one Interferon-related developmental regulator 1 (IFRD1) polynucleotide and/or at least one encoded product thereof, comprising:
- administering to a patient a therapeutically effective dose of at least one antisense oligonucleotide that binds to a natural antisense sequence of said at least one IFRD1 polynucleotide and modulates expression of said at least one IFRD1 polynucleotide;
- thereby preventing or treating the disease associated with the at least one IFRD1 polynucleotide and/or at least one encoded product thereof.
A method of identifying and selecting at least one oligonucleotide selective for a natural antisense transcript of an IFRD1 gene as a selected target polynucleotide for in vivo administration comprising:
- identifying at least one oligonucleotide comprising at least five consecutive nucleotides which are at least partially complementary to a polynucleotide that is antisense to the selected target polynucleotide;
- measuring the thermal melting point of a hybrid of an antisense oligonucleotide and the target polynucleotide or the polynucleotide that is antisense to the selected target polynucleotide under stringent hybridization conditions;
- and selecting at least one oligonucleotide for in vivo administration based on the information obtained.
Dependencies are inferred from the claim text.