RNA-INTERFERENCE BY SINGLE-STRANDED RNA MOLECULES

• US 20150096064A1
• Filed: 07/22/2014
• Published: 04/02/2015
• Est. Priority Date: 07/10/2002
• Status: Active Grant

• Current Assignee: Max-Planck-Gesellschaft Zur Foerderung Der Wissenschaften e.V. (Max-Planck-Gesellschaft zur Frderung der Wissenschaften e)
• Original Assignee: Max-Planck-Gesellschaft Zur Foerderung Der Wissenschaften e.V. (Max-Planck-Gesellschaft zur Frderung der Wissenschaften e)
• Inventors: TUSCHL, Thomas, MARTINEZ, Javier, PATKANIOWSKA, Agnieszka, URLAUB, Henning, LUEHRMANN, Reinhard
• Granted Patent: US 9,476,044 B2
• Time in Patent Office: Days
• US Class Current: 800/13
• CPC Class Codes: A61K 47/549 Sugars, nucleosides, nucleotides or nucleic acids
• CPC Class Codes: A61K 47/557 the modifying agent being biotin
• CPC Class Codes: C07K 14/4705 stimulating, promoting or activating activity
• CPC Class Codes: C07K 19/00 Hybrid peptides , i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
• CPC Class Codes: C12N 15/111 General methods applicable to biologically active non-coding nucleic acids
• CPC Class Codes: C12N 15/113 Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing when used in plants C12N15/8218
• CPC Class Codes: C12N 2310/14 interfering N.A.
• CPC Class Codes: C12N 2310/351 Conjugate
• CPC Class Codes: C12N 2310/3513 Protein; Peptide
• CPC Class Codes: C12N 2320/30 Special therapeutic applications
• CPC Class Codes: C12N 2320/51 modulating the chemical stability, e.g. nuclease-resistance
• CPC Class Codes: C12N 2330/30 chemically synthesised

Abstract

The present invention relates to sequence and structural features of single-stranded (ss)RNA molecules required to mediate target-specific nucleic acid modifications by RNA-interference (RNAi), such as target mRNA degradation and/or DNA methylation.

Claims

16-1. The RISC according to claim 16, wherein said sulfur replaced monophosphate, diphosphate or triphosphate is 5′

-alpha-thiotriphosphate or 5′

-gamma-thiotriphosphate, said 5′

-alkylphosphonate is RP(OH)(O)-0-5′

- or (OH)2(O)P-5′

-CH₂—

), and said 5′

-alkyletherphosphonate is RP(OH)(O)—

O-5′

-.

17-2. The RISC according to claim 1, wherein said RISC has a molecular weight of 120-160 kDa.