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US 5,707,835 AGranted
Ribozymes
AssigneeGene Shears Pty. Limited
InventorsJames Phillip Haseloff, Wayne Lyle Gerlach, Philip Anthony Jennings, Fiona Helen Cameron
Life of this patent
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Abstract
Compounds having highly specific endoribonuclease activity are described. The compounds of this invention, also known as ribozymes, comprise ribonucleotides having two hybridizing regions with predetermined sequences capable of hybridizing with a plant, animal or viral target RNA, a region of defined sequence and a base paired stem region.
Claims11
A compound having the formula:
- 3'----(Y).sub.r --Q--(Y).sub.s --!.sub.z --5'
- wherein each Q represents the structure ##STR10## wherein each X represents a ribonucleotide which may be the same or different;
- wherein each of (X).sub.n and (X).sub.n' represents an oligoribonucleotide having a predetermined sequence which hybridizes with an RNA target sequence to be cleaved, and (b) does not naturally occur covalently bound to the sequences A--A--A--G--C-- and X--C--U--G--A--, respectively, such RNA target sequence not being present within the compound;
- wherein each of n and n' represents an integer which defines the number of ribonucleotides in the oligoribonucleotide with the proviso that the sum of n+n' is greater than 6 and sufficient to allow the compound to stably interact with the RNA target sequence through base pairing;
- wherein each * represents base pairing between the nucleotides located on either side thereof;
- wherein each solid line represents a chemical linkage providing covalent bonds between the ribonucleotides located on either side thereof;
- wherein a represents an integer which defines a number of ribonucleotides with the proviso that a may be 0 or 1 and if 0, the A located 5' of (X).sub.a is bonded to the G located 3' of (X).sub.a ;
- wherein each of m and m' represents an integer which is greater than or equal to 1;
- wherein each of the dashed lines independently represents either a chemical linkage providing covalent bonds between the ribonucleotides located on either side thereof or the absence of any such chemical linkage;
- wherein (X).sub.b represents an oligoribonucleotide which may be present or absent with the proviso that b represents an integer which is greater than or equal to 4 if (X).sub.b is present;
- wherein one or more of the ribonucleotides may be a ribonucleotide derivative;
- wherein each Q may independently be the same or different;
- wherein each Y represents a ribonucleotide which may be the same or different;
- wherein each of r and s represents an integer greater than or equal to 0;
- and
- wherein z represents an integer greater than or equal to 1.
13 more elements
A compound having the formula:
- 3'----(Y).sub.r --Q--(Y).sub.s --!.sub.z --5'
- wherein each Q represents the structure ##STR11## wherein each X represents a ribonucleotide which may be the same or different;
- wherein each of (X).sub.n and (X).sub.n' represents an oligoribonucleotide having a predetermined sequence which hybridizes with an RNA target sequence to be cleaved, and (b) does not naturally occur covalently bound to the sequences A--A--A--G--C-- and X--C--U--G--A--, respectively, such RNA target sequence not being present within the compound;
- wherein each of n and n' represents an integer which defines the number of ribonucleotides in the oligoribonucleotide with the proviso that the sum of n+n' is greater than 6 and sufficient to allow the compound to stably interact with the RNA target sequence through base pairing;
- wherein each * represents base pairing between the nucleotides located on either side thereof;
- wherein each solid line represents a chemical linkage providing covalent bonds between the ribonucleotides located on either side thereof;
- wherein a represents an integer which defines a number of ribonucleotides with the proviso that a may be 0 or 1 and if 0, the A located 5' of (X).sub.a is bonded to the G located 3' of (X).sub.a ;
- wherein each of m and m' represents an integer which is greater than or equal to 3;
- wherein each of the dashed lines independently represents either a chemical linkage providing covalent bonds between the ribonucleotides located on either side thereof or the absence of any such chemical linkage;
- wherein (X).sub.b represents an oligoribonucleotide which may be present or absent with the proviso that b represents an integer which is greater than or equal to 4 if (X).sub.b is present;
- wherein one or more of the ribonucleotides may be a ribonucleotide derivative;
- wherein each Q may independently be the same or different;
- wherein each Y represents a ribonucleotide which may be the same or different;
- wherein each of r and s represents an integer greater than or equal to 0;
- and
- wherein z represents an integer greater than or equal to 1.
13 more elements
Dependencies are inferred from the claim text.