Nucleic Acid Complex
First Claim
Patent Images
1. A complex utilizing one or more functional entities (FEs), said FE(s) being capable of preventing degradation and/or removal of said complex, increasing the activity of said complex and/or increasing the transfer of said complex;
- extracellularly (within an organism), transcellularly (across a cellular membrane) and/or intracellularly (into different locations within a cell), characterized in that said complex comprises a short interfering RNA (siRNA) molecule, wherein said siRNA molecule comprises a first and a second strand and said siRNA molecule is modified in the following way;
at least one anchor-binding domain is incorporated into any one of the two strands of the siRNA molecule, said anchor-binding domain being a nucleic acid or analog thereof,at least one anchor sequence is hybridized to said anchor-binding domain, said anchor sequence being a nucleic acid or analog thereof, andat least one functional entity (FE) is linked to said at least one anchor sequence, said FE(s) being one or more biologically active molecule(s).
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Abstract
The present invention relates to modification of nucleic acids for specific delivery in vitro and in vivo. More specifically, the present invention relates to modification of RNA or DNA molecules in order to add functions in terms of delivery and specificity to RNA interference or antisense technology. A specific binding domain is incorporated into the nucleic acid to which a complementary nucleic acid, conjugated to a biologically active molecule, can hybridize.
35 Citations
63 Claims
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1. A complex utilizing one or more functional entities (FEs), said FE(s) being capable of preventing degradation and/or removal of said complex, increasing the activity of said complex and/or increasing the transfer of said complex;
- extracellularly (within an organism), transcellularly (across a cellular membrane) and/or intracellularly (into different locations within a cell), characterized in that said complex comprises a short interfering RNA (siRNA) molecule, wherein said siRNA molecule comprises a first and a second strand and said siRNA molecule is modified in the following way;
at least one anchor-binding domain is incorporated into any one of the two strands of the siRNA molecule, said anchor-binding domain being a nucleic acid or analog thereof, at least one anchor sequence is hybridized to said anchor-binding domain, said anchor sequence being a nucleic acid or analog thereof, and at least one functional entity (FE) is linked to said at least one anchor sequence, said FE(s) being one or more biologically active molecule(s). - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 62)
- extracellularly (within an organism), transcellularly (across a cellular membrane) and/or intracellularly (into different locations within a cell), characterized in that said complex comprises a short interfering RNA (siRNA) molecule, wherein said siRNA molecule comprises a first and a second strand and said siRNA molecule is modified in the following way;
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22. A method for making a complex that utilises one or more functional entities (FEs), said FE(s) being capable of preventing degradation and/or removal of said complex, increasing the activity of said complex and/or increasing the transfer of said complex;
- extracellularly (within an organism), transcellularly (across a cellular membrane) and/or intracellularly (into different locations within a cell), said complex comprises a short interfering RNA (siRNA) molecule and said siRNA molecule comprises a first and a second strand and wherein the method comprises the following steps;
introducing at least one anchor-binding domain into the siRNA molecule, said anchor-binding domain being a nucleic acid or analog thereof, hybridizing at least one anchor sequence to said anchor-binding domain, said anchor sequence being a nucleic acid or analog thereof, and linking at least one functional entity (FE) to said at least one anchor sequence, said FE(s) being one or more biologically active molecule(s).
- extracellularly (within an organism), transcellularly (across a cellular membrane) and/or intracellularly (into different locations within a cell), said complex comprises a short interfering RNA (siRNA) molecule and said siRNA molecule comprises a first and a second strand and wherein the method comprises the following steps;
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23. A complex utilizing one or more functional entities (FEs), said FE(s) being capable of preventing degradation and/or removal of said complex, increasing the activity of said complex and/or increasing the transfer of said complex;
- extracellularly (within an organism), transcellularly (across a cellular membrane) and/or intracellularly (into different locations within a cell), characterized in that said complex comprises a short hairpin RNA (shRNA) molecule, wherein said shRNA molecule has a double stranded region and a hairpin loop and said shRNA molecule is modified in the following way;
at least one anchor-binding domain is incorporated into the shRNA molecule, said anchor-binding domain being a nucleic acid or analog thereof, at least one anchor sequence is hybridized to said anchor-binding domain, said anchor sequence being a nucleic acid or analog thereof, and at least one functional entity (FE) is linked to said at least one anchor sequence, said FE(s) being one or more biologically active molecule(s). - View Dependent Claims (24, 25, 26, 27, 28, 29, 30, 31, 32, 33, 34, 35, 36, 37, 38, 39, 40, 41, 42, 43, 44)
- extracellularly (within an organism), transcellularly (across a cellular membrane) and/or intracellularly (into different locations within a cell), characterized in that said complex comprises a short hairpin RNA (shRNA) molecule, wherein said shRNA molecule has a double stranded region and a hairpin loop and said shRNA molecule is modified in the following way;
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45. A method for making a complex that utilises functional entities (FEs), said FE(s) being capable of preventing degradation and/or removal of said complex, increasing the activity of said complex and/or increasing the transfer of said complex;
- extracellularly (within an organism), transcellularly (across a cellular membrane) and/or intracellularly (into different locations within a cell), said complex comprises a short hairpin RNA (shRNA) molecule and said shRNA molecule has a double stranded region and a hairpin loop, wherein the method comprises the following steps;
introducing at least one anchor-binding domain into the shRNA molecule, said anchor-binding domain being a nucleic acid or analog thereof, hybridizing at least one anchor sequence to said anchor-binding domain, said anchor sequence being a nucleic acid or analog thereof, and linking at least one functional entity (FE) to said at least one anchor sequence, said FE(s) being one or more biologically active molecule(s).
- extracellularly (within an organism), transcellularly (across a cellular membrane) and/or intracellularly (into different locations within a cell), said complex comprises a short hairpin RNA (shRNA) molecule and said shRNA molecule has a double stranded region and a hairpin loop, wherein the method comprises the following steps;
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46. A complex utilizing one or more functional entities (FEs), said FE(s) being capable of preventing degradation and/or removal of said complex, increasing the activity of said complex and/or increasing the transfer or said complex;
- extracellularly (within an organism), transcellularly (across a cellular membrane) and/or intracellularly (into different locations within a cell), characterized in that said complex comprises an AS (antisense) molecule, wherein said AS molecule is modified in the following way;
at least one anchor-binding domain is incorporated or attached to the AS molecule, said anchor-binding domain being a nucleic acid or analog thereof, at least one anchor sequence is hybridized to said anchor-binding domain, said anchor sequence being a nucleic acid or analog thereof, and at least one functional entity (FE) is linked to said at least one anchor sequence, said FE(s) being one or more biologically active molecule(s). - View Dependent Claims (47, 48, 49, 50, 51, 52, 53, 54, 55, 56, 57, 58, 59, 60)
- extracellularly (within an organism), transcellularly (across a cellular membrane) and/or intracellularly (into different locations within a cell), characterized in that said complex comprises an AS (antisense) molecule, wherein said AS molecule is modified in the following way;
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61. A method for making a complex that utilises functional entities (FEs), said FE(s) capable of preventing degradation and/or removal of said complex, increasing the activity of said complex and/or increasing the transfer of said complex;
- extracellularly (within an organism), transcellularly (across a cellular membrane) and/or intracellularly (into different locations within a cell) and said complex comprises an AS (antisense) molecule, wherein the method comprises the following steps;
incorporating or attaching at least one anchor-binding domain to the AS molecule, said anchor-binding domain being a nucleic acid or analog thereof, hybridizing at least one anchor sequence is to said anchor-binding domain, said anchor sequence being a nucleic acid or analog thereof, and linking at least one functional entity (FE) to said at least one anchor sequence, said FE(s) being one or more biologically active molecule(s).
- extracellularly (within an organism), transcellularly (across a cellular membrane) and/or intracellularly (into different locations within a cell) and said complex comprises an AS (antisense) molecule, wherein the method comprises the following steps;
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63. A kit comprising components for producing a complex capable of transferring one or more siRNA, shRNA or AS molecules across a cellular membrane (intracellularly, extracellularly and/or transcellularly) and into different locations within a cell, said kit comprising at least one siRNA, shRNA and/or AS molecule, at least one anchor-binding domain, at least one anchor sequence being able to hybridize to said anchor-binding domain, and at least one functional entity (FE) linked to said anchor-binding domain, said FE(s) being one or more biologically active molecule(s).
Specification