公开(公告)号：EP1879624A4

公开(公告)日：2009-03-25

申请号：EP06759081

申请日：2006-05-02

申请人： GENZYME CORP

发明人： PASSINI MARCO A ,  DODGE JAMES ,  STEWART GREGORY R

IPC分类号： A61K48/00 ,  C12N15/63 ,  C12N15/74 ,  C12N15/86

CPC分类号： C12N15/86 ,  A61K48/00 ,  C12N2750/14143

摘要： The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (CNS). These disorders include neurometabolic disorders such as lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. They also include disorders such as Alzheimer's disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site.

公开(公告)号：EP2424991A4

公开(公告)日：2012-11-14

申请号：EP10772363

申请日：2010-04-27

申请人： GENZYME CORP

发明人： PASSINI MARCO A ,  SHIHABUDDIN LAMYA ,  CHENG SENG H

IPC分类号： C12N15/79 ,  A61K31/7088 ,  A61K35/76 ,  C07K14/475 ,  C12N15/864

CPC分类号： A61K38/1709 ,  A61K31/7088 ,  A61K48/00 ,  A61K48/0008 ,  A61K48/005 ,  A61K48/0075 ,  C07K14/4702 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14121 ,  C12N2750/14133 ,  C12N2750/14143 ,  C12N2750/14171

摘要： Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.

公开(公告)号：EP1986661A4

公开(公告)日：2012-04-25

申请号：EP07763560

申请日：2007-02-08

申请人： GENZYME CORP

发明人： PASSINI MARCO A ,  ZIEGLER ROBIN J ,  DODGE JAMES ,  SHIHABUDDIN LAMYA ,  CHENG SENG H

IPC分类号： A61K31/70 ,  A61K48/00 ,  C12N9/16 ,  C12N15/85 ,  C12N15/864

CPC分类号： C12N9/16 ,  A01K2207/00 ,  A01K2227/105 ,  A01K2267/0306 ,  A61K48/0083 ,  C12N15/8509 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2840/007 ,  G01N2500/00

摘要： This disclosure pertains to methods and compositions for tolerizing a mammal's brain to exogenously administered acid sphingomyelinase polypeptide by first delivering an effective amount of a transgene encoding the polypeptide to the mammal's hepatic tissue and then administering an effective amount of the transgene to the mammal's central nervous system (CNS).