SINGLE-STRANDED NUCLEIC ACID MOLECULES AND COMPOSITIONS FOR INDUCING A -1 FRAMESHIFT

To provide substances that are safe for living organisms and capable of expressing a protein having an amino acid sequence more similar to that of a normal protein in the state that the length is suppressed from being deleted even from a gene with an out-of-frame mutation, and to provide composition...

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Bibliographic Details
Main Author YANO TAKAMITSU
Format Patent
LanguageEnglish
Japanese
Published 23.03.2023
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Summary:To provide substances that are safe for living organisms and capable of expressing a protein having an amino acid sequence more similar to that of a normal protein in the state that the length is suppressed from being deleted even from a gene with an out-of-frame mutation, and to provide compositions containing the substance.SOLUTION: The foregoing problem is solved by a single-stranded nucleic acid molecule containing a first sequence complementary to a target sequence of a gene of interest and a second sequence forming a stem-loop structure, in the direction from the 5'end side to the 3'end side; and a composition or the like comprising the single-stranded nucleic acid molecule as an active ingredient.SELECTED DRAWING: Figure 7 【課題】本発明の目的は、アウトオブフレーム変異が生じた遺伝子からも、正常なタンパク質とより近似したアミノ酸配列を有するタンパク質を、その長さが欠失することを抑えた状態で発現することができる、生体に対して安全性のある物質及び該物質を含む組成物を提供することにある。【解決手段】上記目的は、5'末端側から3'末端側への方向に、対象となる遺伝子の標的配列に相補的な第1の配列と、ステムループ構造を形成する第2の配列とを含む1本鎖核酸分子;及び該1本鎖核酸分子を有効成分として含む組成物などにより解決される。【選択図】図7
Bibliography:Application Number: JP20210148033