公开(公告)号：WO2016187017A1

公开(公告)日：2016-11-24

申请号：PCT/US2016/032392

申请日：2016-05-13

申请人： MCIVOR, R. Scott ,  BELUR, Lalitha R. ,  KOZARSKY, Karen

发明人： MCIVOR, R. Scott ,  BELUR, Lalitha R. ,  KOZARSKY, Karen

IPC分类号： A61K38/47 ,  C12N7/00 ,  C12N15/86

CPC分类号： A61K38/47 ,  A61K31/675 ,  A61K45/06 ,  A61K48/0075 ,  A61K48/0083 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12Y302/01076 ,  A61K2300/00

摘要： A method to prevent, inhibit or treat one or more symptoms associated with disease of the central nervous system by intranasally, intrathecally, intracerebrovascularly or intravenously administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease, in an amount effective, e.g., to provide for cross-correction.

摘要翻译： 通过鼻内，鞘内，脑内血管内或静脉内施用编码与该疾病相关的基因产物的rAAV，例如基因产物是基因产物的哺乳动物来预防，抑制或治疗与中枢神经系统疾病相关的一种或多种症状的方法 缺乏或以相对于没有疾病的哺乳动物降低的水平存在，其数量有效，例如提供交叉校正。

公开(公告)号：WO2018093925A1

公开(公告)日：2018-05-24

申请号：PCT/US2017061838

申请日：2017-11-15

申请人： LAOHARAWEE KANUT ,  PODETZ PEDERSEN KELLY M ,  KOZARSKY KAREN ,  MCIVOR R SCOTT ,  BELUR LALITHA R

发明人： LAOHARAWEE KANUT ,  PODETZ-PEDERSEN KELLY M ,  KOZARSKY KAREN ,  MCIVOR R SCOTT ,  BELUR LALITHA R

IPC分类号： C12N15/861 ,  A61K38/46 ,  A61K48/00

CPC分类号： A61K38/465 ,  A61K48/00 ,  C12N15/861 ,  C12N2320/32

摘要： A method to prevent, inhibit or treat one or more neurological symptoms associated with a central nervous system disorder, e.g. MPSI or MPSII by, for example, intrathecally, intracerebroventricularly or intravenously administering a rAAV encoding a gene product associated with the disease, e.g., administering to an adult mammal in which the gene product is absent, defective or present at a reduced level relative to a mammal without the disease.

公开(公告)号：WO2022170082A1

公开(公告)日：2022-08-11

申请号：PCT/US2022/015294

申请日：2022-02-04

申请人： REGENTS OF THE UNIVERSITY OF MINNESOTA ,  REGENXBIO INC. ,  MCIVOR, R. Scott ,  BUSS, Nicholas Alexander Piers Sascha ,  LUND, Troy C. ,  BRAUNLIN, Elizabeth ,  BELUR, Lalitha R. ,  FAIRBANKS, Carolyn ,  NEVORET, Marie-Laure

发明人： MCIVOR, R. Scott ,  BUSS, Nicholas Alexander Piers Sascha ,  LUND, Troy C. ,  BRAUNLIN, Elizabeth ,  BELUR, Lalitha R. ,  FAIRBANKS, Carolyn ,  NEVORET, Marie-Laure

IPC分类号： C12N15/864 ,  A61K38/47 ,  A61K48/00 ,  C12N7/00

摘要： A method to prevent, inhibit the progression of, reduce the severity of, or treat cardiac, vascular or skeletal dysfunction or defect(s) in a human having lysosomal storage disorder, is provided.