公开(公告)号：US20130202633A1

公开(公告)日：2013-08-08

申请号：US13760907

申请日：2013-02-06

申请人： Saint Louis University, a non-profit organization

发明人： Adriana Montaño-Suarez ,  Angela Sosa-Molano ,  Alan Knutsen ,  Clifford Bellone ,  Shunji Tomatsu ,  Luis Barrera

IPC分类号： C12N9/16

CPC分类号： C12N9/16 ,  A61K39/001 ,  A61K2039/542 ,  C12Y301/06004

摘要： Disclosed are methods and compositions for determining immunodominant peptides of target enzymes used in enzyme replacement therapy for lysosomal storage disorders. More specifically disclosed are immunodominant peptides for N-acetylgalactosamine-6-sulfatase (GALNS). Also disclosed are methods of inducing oral tolerance towards a target enzyme through oral administration of immunodominant peptides prior to commencing enzyme replacement therapy. More specifically disclosed is a method of inducing oral tolerance for GALNS, by orally administering specific immunodominant peptides for GALNS; in subjects suffering from mucopolysaccharidosis type IVA prior to commencing enzyme replacement therapy using GALNS.

摘要翻译： 公开了用于确定用于溶酶体储存障碍的酶替代疗法中的靶酶的免疫显性肽的方法和组合物。 更具体地公开了N-乙酰半乳糖胺-6-硫酸酯酶（GALNS）的免疫显性肽。 还公开了在开始酶替代治疗之前通过口服施用免疫显性肽来诱导对靶酶的口服耐受性的方法。 更具体地公开了通过口服给予GALNS的特异性免疫显性肽来诱导GALNS的口服耐受性的方法; 在使用GALNS进行酶替代治疗之前，患有IVA型粘多糖症的受试者。