公开(公告)号：US20120258093A1

公开(公告)日：2012-10-11

申请号：US13390236

申请日：2010-08-19

Applicant: Gillian Butler-Browne ,  Suse Dayse Silva-Barbosa ,  Wilson Savino ,  Alexandra Prufer De Queiroz Campos Araujo ,  Fernanda Pinto-Mariz ,  Luciana Rodrigues Carvalho ,  Thomas Voit

Inventor： Gillian Butler-Browne ,  Suse Dayse Silva-Barbosa ,  Wilson Savino ,  Alexandra Prufer De Queiroz Campos Araujo ,  Fernanda Pinto-Mariz ,  Luciana Rodrigues Carvalho ,  Thomas Voit

IPC: G01N21/64 ,  C12Q1/68 ,  A61K31/713 ,  G01N33/566 ,  A61K39/395 ,  A61K31/7088 ,  C40B30/04 ,  A61K38/02

CPC classification number: C07K16/2842 ,  A61K2039/505 ,  G01N33/56972 ,  G01N2333/7055 ,  G01N2800/2885 ,  G01N2800/52

Abstract: The invention relates to methods for the treatment of Duchenne muscular dystrophy and to methods for determining the prognosis of a subject affected with Duchenne Muscular Dystrophy. More particularly, the present invention relates to a VLA-4 antagonist for use in the treatment of Duchenne Muscular Dystrophy. The present invention also relates to a method for determining the prognosis of a subject affected with Duchenne Muscular Dystrophy wherein said method comprising a step consisting of determining the level of VLA-4 high T cells in a blood sample obtained from said subject.

Abstract translation: 本发明涉及治疗杜氏肌营养不良症的方法和用于确定受杜氏肌营养不良症影响的受试者的预后的方法。 更具体地说，本发明涉及用于治疗杜氏肌营养不良症的VLA-4拮抗剂。 本发明还涉及用于确定受杜氏肌营养不良影响的受试者的预后的方法，其中所述方法包括测定从所述受试者获得的血液样品中VLA-4高T细胞的水平的步骤。

公开(公告)号：US20120172415A1

公开(公告)日：2012-07-05

申请号：US13393004

申请日：2010-08-31

Applicant: Thomas Voit ,  Luis Garcia ,  Valerie Robin ,  Patrick Dreyfus

Inventor： Thomas Voit ,  Luis Garcia ,  Valerie Robin ,  Patrick Dreyfus

IPC: A61K31/7088 ,  A61P9/00 ,  A61P21/00 ,  C12N15/113 ,  C07K14/47

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2320/33 ,  C12N2310/3521

Abstract: Methods for stabilizing unstable proteins or for restoring functionality to non-functional or poorly functioning (semi-functional) proteins using exon skipping technology are provided. The methods involve the administration of antisense oligonucleotides to cause exon skipping, thereby removing one or more exons responsible for protein instability or lack of functionality. For example, exons encoding protease recognition sites may be removed. The method is useful for treating diseases caused by protein instability, such as Becker Muscular Dystrophy, or for treating Duchenne Muscular Distrophy patients with semi-functional dystrophin due to treatment with other exon skipping or stop codon readthrough therapies.

Abstract translation: 提供了使用外显子跳过技术来稳定不稳定蛋白质或恢复非功能或功能不良（半功能）蛋白功能的方法。 所述方法包括给予反义寡核苷酸引起外显子跳跃，从而去除负责蛋白质不稳定或缺乏功能的一个或多个外显子。 例如，可以去除编码蛋白酶识别位点的外显子。 该方法可用于治疗由蛋白质不稳定性引起的疾病，例如贝克肌营养不良症，或用于治疗具有半功能性肌营养不良蛋白的Duchenne肌肉萎缩症患者，这是由于其他外显子跳过或终止密码子阅读疗法治疗。