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1. WO2012133947 - FUNCTIONAL NUCLEIC ACID MOLECULE AND USE THEREOF

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Claims

Claim 1

A functional nucleic acid molecule comprising:

(a) a target determinant sequence comprising antisense sequence to a target sequence in the protein-encoding RNA for which protein synthesis efficiency is to be increased; and

(b) a regulatory sequence having an activity of increasing of the protein synthesis efficiency.

Claim 2

The functional nucleic acid molecule as set forth in claim

1 , wherein the regulatory sequence comprises a SINE (Short Interspersed Element) derived sequence .

Claim 3

The functional nucleic acid molecule as set forth in claim

2 , wherein the SINE derived sequence is a SINE-B2 -derived sequence.

Claim 4

The functional nucleic acid molecule as set forth in claim 1 , wherein :

the regulatory sequence is selected from the group consisting of the following ( 1 ) through (5) :

( 1 ) an RNA, which is encoded by a D NA consisting of the nucleotide sequence shown in SEQ ID No : l

(2) an RNA, which is encoded by a DNA consisting of the nucleotide sequence shown in SEQ ID No : 2

(3) an RNA, which is encoded by a DNA consisting of the nucleotide sequence shown in SEQ ID No : 3

(4) nucleic acids (i) which is at least 25% similarity to the RNA, which is encoded by a DNA consisting of the nucleotide sequence shown in SEQ ID No : 1 , 2 or 3 and (ii) which has a function of increasing the protein synthesis efficiency; and

(5) nucleic acids (i) which is encoded by a DNA in which not less than 1 but not more than 200 nucleotides are deleted, substituted, added, and / or inserted in the nucleotide sequence shown in SEQ ID No : 1 , 2 or 3 and (ii) which has a function of increasing the protein synthesis efficiency.

Claim 5

The functional nucleic acid molecule as set forth in any one of claims 1 to 4 , wherein the target determinant sequence is located between a 5 '-terminal and the regulatory sequence in the functional nucleic acid molecule .

Claim 6

The functional nucleic acid molecule as set forth in any one of claims 1 to 5, wherein the target determinant sequence has a length of 7 nucleotides to 250 nucleotides .

Claim 7

The functional nucleic acid molecule as set forth in any one of claims 1 to 6 , wherein the target determinant sequence has at least 60% similarity to a sequence complementary to a corresponding sequence in the protein-encoding RNA or a sequence around the first 5 '-terminal start codon of the protein-encoding sequence .

Claim 8

The functional nucleic acid molecule as set forth in any one of claims 1 to 7, wherein the regulatory sequence of the functional nucleic acid molecule is oriented in a reverse direction relative to the direction of translation .

Claim 9

The functional nucleic acid molecule as set forth in any one of claims 1 to 8 , wherein the target determinant sequence is designed to be hybridizable with a 5 '-UTR (untranslated region) of the protein-encoding RNA or a sequence around the first 5 '-terminal start codon of the protein-encoding sequence .

Claim 10

A method for producing a functional nucleic acid molecule, comprising the step of preparing an RNA molecule that comprises:

(a) a target determinant sequence comprising antisense sequence to a target sequence in the protein-encoding RNA for which protein synthesis efficiency is to be increased; and

(b) a regulatory sequence having an activity of increasing of the protein synthesis efficiency.

Claim 1 1

A DNA molecule encoding a functional nucleic acid molecule as set forth in any one of claims 1 to 9.

Claim 12

An expression vector comprising a functional nucleic acid molecule as set forth in any one of claims 1 to 9 , or a DNA molecule as set forth in claim 1 1 .

Claim 13

A composition for increasing protein synthesis efficiency, which comprises a functional nucleic acid molecule as set forth in any one of claims 1 to 9 , a DNA molecule as set forth in clam 1 1 , or an expression vector as set forth in claim 12.

Claim 14

A method for increasing the protein synthesis efficiency, comprising the step of:

(a) allowing a functional nucleic acid molecule as set forth in any one of claims 1 to 9 to coexist with a protein-encoding RNA, which partial sequence of the protein-encoding RNA has similarity with the target determinant sequence of the functional RNA molecule .

Claim 15

The method as set forth in claim 14 , wherein :

the step (a) comprises transfecting into a cell the functional nucleic acid molecule or a DNA molecule as set forth in claim 1 1 .

Claim 16

A method for producing a protein, comprising the step of increasing the protein synthesis efficiency by a method for increasing the protein synthesis efficiency as set forth in claim 14 or 15.

Claim 17

A method for treating a disease that is caused by a quantitative decrease in a protein, comprising the step of increasing the protein synthesis efficiency by a method for increasing the protein synthesis efficiency as set forth in claim 14 or 15 in a a subject having the disease or a predisposition to the disease .