TREATMENT OF α-GALACTOSIDASE A DEFICIENCY

• Main Authors: WILLIAMS MELANIE D, SCHUETZ THOMAS J, KINOSHITA CAROL M, DANIEL PETER F, SELDEN RICHARD F, TRECO DOUGLAS A, BOROWSKI MARIANNE
• Format: Patent
• Language: English; Japanese
• Published: 14.05.2015
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; ORGANIC CHEMISTRY; PEPTIDES; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

PROBLEM TO BE SOLVED: To provide a highly refined α-Gal A and various refining methods for the same, to provide an α-Gal A preparation having altered electric charges and a method for producing the same, to provide an α-Gal A preparation having circulatory half-life extended in a mammalian host and a method for producing the same, and to provide a method for dosing such an α-Gal A preparation to a subject and a dosage thereof.SOLUTION: There is provided a composition comprising a human α-Gal A preparation refined to a homogeneity of at least 99.5% when determined by SDS-PAGE or reverse-phase HPLC, in which the preparation contains various α-Gal A glycoforms, has a specific activity of at least 3.0×10units/mg protein, and substantially contains no lectin. 【課題】高度に精製されたα−Ｇａｌ Ａ、およびそれを精製する様々な方法；変更された電荷を有するα−Ｇａｌ Ａ調製品およびその調製品の製造方法；ほ乳類宿主中で延長された循環半減期を有するα−Ｇａｌ Ａ調製品、およびその製造方法；α−Ｇａｌ Ａ調製品を被験者に投与する方法および投与量を提供する。【解決手段】SDS-PAGEまたは逆相HPLCにより測定された場合、少なくとも99.5％の均質性まで精製されたヒトα−Gal A調製物を含む組成物であって、該調製物は、多様なα−Gal Aグリコフォームを含み、少なくとも3.0?106ユニット／ｍｇタンパク質の比活性を有し、かつ、実質的にレクチンを含まない【選択図】なし