КОНСИНО Майкл Ф. (US),КАЛИАС Периклс (US),ПАН Цзинь (US),ХОЛМС Кевин (US),МАРТИНИ Паоло (US),РОМАШКО Алла (US),МЕЙЯППАН Мутураман (US),ЧЖАН Бохун (US),ИСКЕНДЕРИАН Андреа (US),ЛУНДБЕРГ Дианна (US),НОРТ
申请号：
RU2012154832/10
公开号：
RU2012154832A
申请日:
2011.06.25
申请国别(地区):
RU
年份:
2014
代理人:
摘要：
1. A method of treating Sanfilippo disease type B (SanB), comprising the step of intrathecal administration to a subject in need of treatment a recombinant protein alpha-N-acetylglucosaminidase (Naglu) .2. A method according to claim 1, characterized in that said Naglu recombinant protein is a fusion protein comprising Naglu domain and targeting to lysosomes fragment.3. A method according to claim 2, characterized in that said Naglu domain comprises an amino acid sequence at least 80% identical to SEQ ID NO: 1 (the mature protein of human Naglu) .4. A method according to claim 2, characterized in that said Naglu domain comprises an amino acid sequence at least 95% identical to SEQ ID NO: 1 (the mature protein of human Naglu) .5. A method according to claim 2, characterized in that said Naglu domain comprises an amino acid sequence identical to SEQ ID NO: 1 (the mature protein of human Naglu) .6. A method according to claim 2, characterized in that said at lysosome targeting moiety is a fragment of IGF-II.7. A method according to claim 6, characterized in that said IGF-II fragment comprises an amino acid sequence at least 70% identical to mature human IGF-II (SEQ ID NO: 3) .8. A method according to claim 6, characterized in that said IGF-II fragment comprises an amino acid sequence at least 80% identical to mature human IGF-II (SEQ ID NO: 3) .9. A method according to claim 6, characterized in that said IGF-II fragment comprises an amino acid sequence at least 90% identical to mature human IGF-II (SEQ ID NO: 3) .10. A method according to claim 6, characterized in that said IGF-II fragment comprises an amino acid sequence comprising residues 8-67 of mature human IGF-II (SEQ ID NO: 3) .11. A method according to any one p.p.2-10, characterized1. Способ лечения болезни Санфилиппо типа В (SanB), включающий этап интратекального введения нуждающемуся в лечении субъекту рекомбинантного белка альфа-N-ацетилглюкозаминидазы (Naglu).2. Способ по п.1, отличающийся тем, что указанн
