Abstract: RNA molecules for RNA interference to target a mutant allele with a point mutation, wherein the molecule has a nucleotide sequence complementary to a nucleotide sequence of a coding region of the mutant allele; and when counted from the base at the 5?-end in the nucleotide sequence complementary to the sequence of the mutant allele: a base at position 5 or 6 is mismatched with a base in the mutant allele; a base at position 10 or 11 is at the position of the point mutation and is identical to the base at the position of the point mutation in the mutant allele; the group at the 2?-position of the pentose in the ribonucleotide at position 8 is modified with OCH3, halogen, or LNA; and the group at the 2?-position of the pentose in the ribonucleotide at position 7 is not modified with any of OCH3, halogen, and LNA.

Abstract: Described herein are antiviral silencing RNA molecules (siRNAs), pharmaceutical compositions comprising same and uses thereof for the treatment of viral infections. In embodiments the siRNAs comprises chemically modification(s) for enhanced cell penetrating abilities and/or for greater nuclease resistance. Examples of chemical modifications include substituting one or more nucleotides of a native siRNA molecule with 2?-O-Methylnucleoside, 2?-Fluoronucleoside, aminoalkyl-nucleotide, aminoethyl-nucleotide, and/or 5?-aminopropyl-2?-OMe-nucleoside. The chemically modified nucleotides may be incorporated into the P strand, the G strand or both. Other possible modifications include coupling a compound such as a spermine molecule to the 5? terminus or the 3? terminus of the siRNA.

Abstract: RNA molecules for RNA interference to target a mutant allele with a point mutation, wherein the molecule has a nucleotide sequence complementary to a nucleotide sequence of a coding region of the mutant allele; and when counted from the base at the 5?-end in the nucleotide sequence complementary to the sequence of the mutant allele: a base at position 5 or 6 is mismatched with a base in the mutant allele; a base at position 10 or 11 is at the position of the point mutation and is identical to the base at the position of the point mutation in the mutant allele; the group at the 2?-position of the pentose in the ribonucleotide at position 8 is modified with OCH3, halogen, or LNA; and the group at the 2?-position of the pentose in the ribonucleotide at position 7 is not modified with any of OCH3, halogen, and LNA.

Abstract: The present invention is directed to provide novel RNA molecules, chimeric NA molecules, double-stranded RNA molecules, and double-stranded chimeric NA molecules. Specifically, an embodiment of the present invention is an RNA molecule for RNA interference to target a mutant allele with a point mutation, in which (1) the molecule has a nucleotide sequence complementary to a nucleotide sequence of a coding region of the mutant allele; and (2) when counted from the base at the 5?-end in a nucleotide sequence complementary to a nucleotide sequence of the mutant allele, (2-1) a base at position 5 or 6 is mismatched to a base in the mutant allele; (2-2) a position 10 or 11 corresponds to the position of the point mutation; and (2-3) a group at the 2?-position of a pentose at positions 6-8 or positions 7 and 8 is modified with, e.g., OCH3. In this RNA molecule, one or more ribonucleotides may be replaced by, e.g., a deoxyribonucleotide. The molecule may form a double-stranded RNA with a complementary strand.

Abstract: An oligo- or polynucleotide for an RNA interference comprising a sense sequence, a trimming sequence, and an antisense sequence in this order is provided. In an example, the sense sequence is homologous to a part of a sequence of a target gene wherein a base in the nucleotide of 5? end is guanine, and a base in the nucleotide of 3? end is adenine, thymine, or uracil, the antisense sequence is complementary to the sense sequence and in the 7-bp-long region of the 5? terminal at least one base selected from the group consisting of adenine, thymine, and uracil is rich, and the trimming sequence comprises 5 to 52 nucleotides and is represented by the formula: (G or C)—X—Y-Z-(C or G) wherein the sense sequence, the trimming sequence, and the antisense sequence are consecutive, and this consecutive sequence does not comprise four or more consecutive nucleotides of which bases are thymine and/or uracil.

Abstract: The present invention provides an in-vitro-derived component which permits identification of essential components contained in an extracted fraction having physiological activities such as a survival activity on neuron, particularly to spinal cord motoneuron, and is useful for diagnosis, prevention and therapy of various diseases including neurodegenerative diseases. The RNA of the present invention has a base sequence represented by the sequence No. 1 of the sequence table, exhibiting a very high survival activity relative to spinal cord motoneuron.