C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/11 (2006.01) A61K 31/70 (2006.01) C07H 21/02 (2006.01) C12N 9/22 (2006.01) C12N 15/82 (2006.01)
Patent
CA 2193373
This invention is directed to improved catalytic compounds, hammerhead ribozymes, capable of hybridizing with a target RNA to be cleaved. These improved compounds have optimized stems (X)m * (X)m', loops (X)b and hybridizing arms. The invention is also directed to compositions for enhanced RNA cleavage which comprise a first synthetic non-naturally occurring oligonucleotide compound which comprises nucleotides whose sequence defines a conserved catalytic region and nucleotides whose sequence is capable of hybridizing with a predetermined target sequence and a second synthetic non- naturally occurring oligonucleotide which does not contain the predetermined target sequence and is complementary to at least a portion of the first oligonucleotide compound. The invention is also directed to synthetic non- naturally occurring oligonucleotide compounds embedded in a tRNA. The ribozymes and compositions of the present invention may be used in vitro or in vivo. They may be used as diagnostic or therapeutic agents.
La présente invention concerne des composés catalytiques améliorés, des ribozymes à tête de marteau, capables de s'hybrider avec un ARN cible à cliver. Ces composés améliorés portent des tiges (X)m * (X)m', des boucles (X)b et des bras d'hybridation optimisés. L'invention concerne également des compositions qui permettent un clivage amélioré de l'ARN et qui comprennent, d'une part, un premier composé oligonucléotide de synthèse n'existant pas à l'état naturel, qui est constitué de nucléotides dont la séquence est capable de définir une région catalytique conservée et de nucléotides dont la séquence est capable d'hybridation avec une séquence cible prédéterminée, et d'autre part un second composé oligonucléotide de synthèse n'existant pas à l'état naturel, qui ne comporte pas une séquence cible prédéterminée, mais qui est complémentaire d'au moins une partie du premier composé oligonucléotide. L'invention concerne enfin des composés oligonucléotides synthétiques n'existant pas à l'état naturel et encapsulés dans un ARNt. Les ribozymes et compositions de la présente invention peuvent s'utiliser, in vitro et in vivo, comme agents de diagnostics ou agents thérapeutiques.
Keese Paul
Perriman Rhonda
Stapper Marianne
Gene Shears Pty. Ltd.
Gowling Lafleur Henderson Llp
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