ФЕРВЕКЕН Воутер (BE),ПИЕНС Кейтлин Камилла Телефвор Алида Мария (BE),СТАУТ Ян Робет Лудо (BE),ПИНАЭРТ Гвенда Ноэлла (BE)
申请号：
RU2014139953
公开号：
RU2014139953A
申请日:
2013.03.14
申请国别(地区):
RU
年份:
2016
代理人:
摘要：
1. An isolated molecular complex having acid alpha glucosidase (GAA) activity and comprising at least two polypeptides, each polypeptide having at least 85% sequence identity with respect to the amino acid sequence segment indicated in SEQ ID NO: 1, wherein each segment is obtained by proteolysis of the amino acid sequence indicated in SEQ ID NO: 1 at one or more sites between amino acid 50 and amino acid 74； wherein said molecular complex includes at least one modification, which leads to an increase in the ability of said molecular complex to be transported inside a mammalian cell. The molecular complex according to claim 1, characterized in that each segment is obtained by proteolysis of the amino acid sequence indicated in SEQ ID NO: 1 at one or more sites between amino acid 60 and amino acid 65.3. The molecular complex according to claim 1, characterized in that said complex comprises at least three polypeptides. The molecular complex according to any one of paragraphs. 1-3, characterized in that the proteolysis of the amino acid sequence indicated in SEQ ID NO: 1 also includes cleavage at one or more sites between amino acid 719 and amino acid 746 or cleavage at one or more sites between amino acid 137 and amino acid 151 of the amino acid sequence, specified in SEQ ID NO: 1.5. The molecular complex according to paragraphs. 1-3, characterized in that the complex comprises at least four polypeptides. The molecular complex of claim 5, wherein proteolysis also includes cleavage at one or more sites between amino acid 719 and amino acid 746 after1. Выделенный молекулярный комплекс, обладающий активностью кислой альфа-глюкозидазы (GAA) и включающий, по меньшей мере, два полипептида, при этом каждый полипептид обладает, по меньшей мере, 85% идентичностью последовательности относительно сегмента аминокислотной последовательности, указанной в SEQ ID NO:1, при этом каждый сегмент получен протеолизом аминокислотной последовательности, указанной в SEQ ID NO:1 в одном или
