CRYSTAL STRUCTURE OF HUMAN ALPHA-N-ACETYLGLUCOSAMINIDASE
摘要
The present invention provides the three-dimensional structure of human a-N- acetylglucosaminidase (NAGLU) protein. This crystallographic information is useful in the identification and development of novel binding compounds of NAGLU, NAGLU mutants, for example, those associated with Sanfilippo syndrome type B (mucopolysaccharidosis III B (MPS III-B)), and other NAGLU family members (family 89 a-N-acetylglucosaminidase) which may modulate the activity and/or stability of mutated NAGLU. Such compounds may be useful for the treatment of Sanfilippo syndrome type B (mucopolysaccharidosis III B (MPS III-B)).
申请公布号
WO2012012742(A3)
申请公布日期
2012.04.05
申请号
WO2011US45045
申请日期
2011.07.22
申请人
SHIRE HUMAN GENETIC THERAPIES, INC.;MEIYAPPAN, MUTHURAMAN;CONCINO, MICHAEL, F.;NORTON, ANGELA, W.
发明人
MEIYAPPAN, MUTHURAMAN;CONCINO, MICHAEL, F.;NORTON, ANGELA, W.