TREATMENT OF SANFILIPPO SYNDROME TYPE B

• Main Authors: PAN JING, BETTINA STRACK-LOGUE, DIANNA LUNDBERG, MUTHURAMAN MEIYAPPAN, ANGELA NORTON, RICHARD PFEIFER, ZHANG BOHONG, YAN HUANG, PAOLO MARTINI, MICHAEL F CONCINO, PERICLES CALIAS, MARY ALESSANDRINI, ANDREA ISKENDERIAN, KEVIN HOLMES, ALLA ROMASHKO
• Format: Patent
• Language: English; Japanese
• Published: 26.05.2016
• 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 methods and compositions of treating Sanfilippo syndrome type B (Sanfilippo B).SOLUTION: A method of treating Sanfilippo syndrome type B comprises a step of administering intrathecally to a subject in need of treatment a recombinant α-N-acetylglucosaminidase (Naglu) protein. The recombinant Naglu protein is a fusion protein which comprises a Naglu domain and a lysosomal targeting moiety and further comprises a linker between the Naglu domain and the lysosomal targeting moiety.SELECTED DRAWING: Figure 1 【課題】サンフィリポ症候群Ｂ型（サンフィリポＢ）の治療方法および組成物の提供。【解決手段】治療を必要とする対象に組換えα−Ｎ−アセチルグルコサミニダーゼ（Ｎａｇｌｕ）タンパク質を髄腔内投与するステップを含むサンフィリポ症候群Ｂ型の治療方法で、前記組換えＮａｇｌｕタンパク質が、Ｎａｇｌｕドメインとリソソーム標的化部分とを含む融合タンパク質であり、Ｎａｇｌｕドメインとリソソーム標的化部分との間にリンカーをさらに含む。【選択図】図１