Patent ID: 6828148
Filing Date: 2004-12-07
Classification: A61K,C12N

Abstract:
A miniribozyme of the formula IA or IB: wherein each X represents a nucleotide which may be the same or different and may be substituted or modified in its sugar, base or phosphate; and wherein G10.1, and C11.1 each represent a nucleotide which may be substituted or modified in its sugar (which may be ribose or deoxyribose), base or phosphate; wherein each of C, G, A and U represents a ribonucleotide which may be substituted or modified in its sugar, base or phosphate; wherein each of (X)n and (X)n-1-A and (X)nâ€² represents an oligonucleotide having a pre-determined sequence which is capable of hybridizing with an RNA target sequence to be cleaved, such RNA target sequence not being present within the compound, and each of n and nâ€² represents an integer which defines the number of nucleotides in the oligonucleotide; wherein Xâ€² represents a ribonucleotide selected from C, G, A and U which may be substituted or modified in its sugar, base or phosphate; wherein a defines the number of nucleotides in (X)n and may be 0 or 1 and if 0, the A located 5â€² of (X)n is bonded to the G located 3â€² of (X)n; wherein each solid line represents a chemical linkage providing covalent bonds between the nucleotides located on either side thereof; wherein each N represents a nucleotide selected from C, G, A and U/T which may be substituted or modified in its sugar (which may be ribose or deoxyribose), base or phosphate and wherein each H represents a nucleotide selected from C, A and U/T, which may be substituted or modified in its sugar (which may be ribose or deoxyribose), base or phosphate; with the proviso that the sequence 5â€²-NNHH-3â€² is not UUUU or TTTT, CUCC, MUU or GGCA.