公开(公告)号：US06582692B1

公开(公告)日：2003-06-24

申请号：US09715858

申请日：2000-11-17

申请人： Gregory Podsakoff ,  Gordon Watson

发明人： Gregory Podsakoff ,  Gordon Watson

IPC分类号： A01N6300

CPC分类号： C12N15/86 ,  A61K48/00 ,  C12N9/2434 ,  C12N2750/14143 ,  C12N2830/008 ,  C12N2830/15 ,  C12N2830/42 ,  C12N2830/85 ,  C12N2840/44 ,  C12Y302/01031

摘要： AAV expression vectors and recombinant virions produced using these vectors, which include genes coding for enzymes defective or missing in lysosomal storage disorders, are described. These recombinant AAV virions are useful in the treatment of a variety of lysosomal storage disorders and the methods described herein provide for long-term, sustained expression of the defective or missing enzyme.

公开(公告)号：US20060104954A1

公开(公告)日：2006-05-18

申请号：US11329941

申请日：2006-01-11

申请人： Gregory Podsakoff ,  Gordon Watson ,  Linda Couto ,  Bin Yang

发明人： Gregory Podsakoff ,  Gordon Watson ,  Linda Couto ,  Bin Yang

IPC分类号： A61K48/00

CPC分类号： C12N15/86 ,  A61K48/00 ,  C12N9/2434 ,  C12N2750/14143 ,  C12N2830/008 ,  C12N2830/15 ,  C12N2830/42 ,  C12N2830/85 ,  C12N2840/44 ,  C12Y302/01031

摘要： AAV expression vectors and recombinant virions produced using these vectors, which include genes coding for enzymes defective or missing in lysosomal storage disorders, are described. These recombinant AAV virions are useful in the treatment of a variety of lysosomal storage disorders and the methods described herein provide for long-term, sustained expression of the defective or missing enzyme.

公开(公告)号：US20060099185A1

公开(公告)日：2006-05-11

申请号：US11305629

申请日：2005-12-15

申请人： Gregory Podsakoff ,  Gary Kurtzman

发明人： Gregory Podsakoff ,  Gary Kurtzman

IPC分类号： A61K48/00

CPC分类号： C12N15/86 ,  A01K2217/05 ,  A61K38/1816 ,  A61K38/47 ,  A61K48/00 ,  A61K48/0058 ,  A61K48/0075 ,  C07K14/505 ,  C12N9/2402 ,  C12N2750/14143 ,  C12N2830/42 ,  C12N2840/44 ,  C12Y302/0102 ,  C12Y302/01023 ,  Y10S514/814 ,  Y10S514/815

摘要： The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue in the treatment of anemia is disclosed, The invention allows for the direct, in vivo injection of recombinant AAV virions into muscle tissue, e.g., by intramuscular injection, as well as for the in vitro transduction of muscle cells which can subsequently be introduced into a subject for treatment. The invention provides for sustained, high-level expression of a delivered nucleotide sequence encoding erythropoietin, and for in vivo secretion thereof from transduced muscle cells such that systemic delivery is achieved.

摘要翻译： 公开了使用重组腺相关病毒（AAV）病毒粒子将DNA分子递送到肌肉细胞和组织以治疗贫血症。本发明允许将重组AAV病毒体直接体内注射入肌肉组织，例如， 通过肌内注射，以及肌肉细胞的体外转导，随后可以将其引入受试者进行治疗。 本发明提供了编码促红细胞生成素的递送核苷酸序列的持续高水平表达，以及用于体内从转导的肌肉细胞分泌，从而实现全身递送。

公开(公告)号：US20080069803A1

公开(公告)日：2008-03-20

申请号：US11980214

申请日：2007-10-30

申请人： Gregory Podsakoff ,  Paul Kessler ,  Barry Byrne ,  Gary Kurtzman

发明人： Gregory Podsakoff ,  Paul Kessler ,  Barry Byrne ,  Gary Kurtzman

IPC分类号： A61K48/00 ,  C12N15/63 ,  C12N15/861 ,  C12N5/00 ,  C12N7/01

CPC分类号： C12N15/86 ,  A01K2217/05 ,  A61K38/1816 ,  A61K38/47 ,  A61K48/00 ,  A61K48/0075 ,  C07K14/505 ,  C12N9/2408 ,  C12N2750/14143 ,  C12N2799/025 ,  C12N2830/42 ,  C12N2840/44 ,  C12Y302/0102 ,  C12Y302/01023

摘要： The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue is disclosed. The invention allows for the direct, in vivo injection of recombinant AAV virions into muscle tissue, e.g., by intramuscular injection, as well as for the in vitro transduction of muscle cells which can subsequently be introduced into a subject for treatment. The invention provides for sustained, high-level expression of the delivered gene and for in vivo secretion of the therapeutic protein from transduced muscle cells such that systemic delivery is achieved.

摘要翻译： 公开了使用重组腺相关病毒（AAV）病毒颗粒将DNA分子递送至肌肉细胞和组织。 本发明允许将重组AAV病毒体直接，体内注射到肌肉组织中，例如通过肌内注射，以及肌肉细胞的体外转导，随后可将其导入受试者进行治疗。 本发明提供所传递的基因的持续高水平表达以及用于体内分泌来自转导的肌细胞的治疗性蛋白质，从而实现全身递送。

公开(公告)号：US20060099184A1

公开(公告)日：2006-05-11

申请号：US11303896

申请日：2005-12-15

申请人： Gregory Podsakoff ,  Paul Kessler ,  Barry Byrne ,  Gary Kurtzman

发明人： Gregory Podsakoff ,  Paul Kessler ,  Barry Byrne ,  Gary Kurtzman

IPC分类号： A61K48/00 ,  C12N15/861

CPC分类号： C12N15/86 ,  A01K2217/05 ,  A61K38/1816 ,  A61K38/47 ,  A61K48/00 ,  A61K48/0075 ,  C07K14/505 ,  C12N9/2408 ,  C12N2750/14143 ,  C12N2799/025 ,  C12N2830/42 ,  C12N2840/44 ,  C12Y302/0102 ,  C12Y302/01023

摘要： The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue is disclosed. The invention allows for the direct, in vivo injection of recombinant AAV virions into muscle tissue, e.g., by intramuscular injection, as well as for the in vitro transduction of muscle cells which can subsequently be introduced into a subject for treatment. The invention provides for sustained, high-level expression of the delivered gene and for in vivo secretion of the therapeutic protein from transduced muscle cells such that systemic delivery is achieved.

摘要翻译： 公开了使用重组腺相关病毒（AAV）病毒颗粒将DNA分子递送至肌肉细胞和组织。 本发明允许将重组AAV病毒体直接，体内注射到肌肉组织中，例如通过肌内注射，以及肌肉细胞的体外转导，随后可将其导入受试者进行治疗。 本发明提供所传递的基因的持续高水平表达以及用于体内分泌来自转导的肌细胞的治疗性蛋白质，从而实现全身递送。