PROBLEM TO BE SOLVED: To provide methods and compositions for modulating sialic acid production in a system, as well as methods and compositions for treating and/or preventing Hereditary Inclusion Body Myopathy and/or symptoms thereof.SOLUTION: Methods for modulating the production of sialic acid in a system are provided, which comprise providing the system with a wild-type GNE-encoding nucleic acid sequence, wherein the system comprises a cell, muscular tissue, or other desirable targets. The present invention encompasses methods for producing wild-type GNE in a system that comprises a mutated endogenous GNE-encoding sequence, which methods comprise providing, for example, a cell or muscular tissue that harbors a mutated (defective) GNE-encoding sequence with a functional wild-type GNE encoding sequence. Also provided is a method for modulating the production of sialic acid in a system, which comprises providing the system with a wild-type GNE-encoding nucleic acid sequence comprising a promoter being the CMV promoter.【課題】系の中でのシアル酸の産生を調節する方法及び組成物、並びに遺伝性封入体ミオパチー及び／又はその症状を治療及び／又は予防する方法及び組成物の提供。【解決手段】細胞、筋組織又は他の望ましい標的を含む系に、野生型ＧＮＥコード核酸配列を与えるステップを含む系中でのシアル酸の産生を調節する方法であって、変異型内因性ＧＮＥコード配列を含む系の中で野生型ＧＮＥを産生する方法を包含し、機能的な野生型ＧＮＥコード配列を、例えば、変異型（欠陥を有する）ＧＮＥコード配列を有する細胞又は筋組織に与えるステップを含む方法。系中でのシアル酸の産生を調節する方法であって、プロモーターがＣＭＶプロモーターである、前記系に野生型ＧＮＥコード核酸配列を与えるステップを含む方法、【選択図】なし
