公开(公告)号：US20050048039A1

公开(公告)日：2005-03-03

申请号：US10766929

申请日：2004-01-30

申请人： Patrick Dreyfus ,  Elaine Parrish ,  Luis Garcia ,  Mohamed Chokri ,  Jacques Bartholeyns ,  Elise Peltekian

发明人： Patrick Dreyfus ,  Elaine Parrish ,  Luis Garcia ,  Mohamed Chokri ,  Jacques Bartholeyns ,  Elise Peltekian

IPC分类号： C12N15/09 ,  A61K35/14 ,  A61K38/00 ,  A61K48/00 ,  A61P9/10 ,  A61P11/00 ,  A61P19/02 ,  A61P19/08 ,  A61P21/00 ,  A61P25/00 ,  A61P25/02 ,  C12N5/0786 ,  C12N5/10 ,  G01N33/569 ,  G01N33/68

CPC分类号： C12N5/0645 ,  A61K38/00 ,  A61K48/00 ,  C12N2510/00 ,  G01N33/56972 ,  G01N33/6893 ,  G01N33/6896

摘要： Method for the treatment or diagnosis of pathologies either expressed in injured or pathological multiple sites in tissues or in the body or expressed in injured or pathological sites of tissues or cells in sites of the body, which are difficult to access, with said sites or areas in immediate proximity to said sites being the source of the release of chemotactic factors for endogenous macrophages, either spontaneously or upon suitable stimulation, wherein said treatment is carried out by administration to the body of an appropriate amount of exogenous monocyte derived cells, said monocyte derived cells being, in the case of treatment, loaded with corrective agents with respect to the pathologies to be treated, and with said monocyte derived cells having the properties of mobilisation towards the source of the above-said released chemotactic factors and in target the cells present in the vicinity of the said released chemotactic factors, and in the case of diagnosis, loaded with a marker enabling the detection of injured or pathological sites.

摘要翻译： 用于治疗或诊断在组织或体内的损伤或病理多个部位中表达的病理学或在身体部位的组织或细胞的损伤或病理部位表达的病症，所述部位或部位难以进入所述部位或区域 紧邻所述位点是内源性巨噬细胞的趋化因子释放的来源，或者是自发地或在适当的刺激下，其中所述的治疗通过向人体施用适量的外源性单核细胞来源的细胞来进行，所述单核细胞来源于 在治疗的情况下，细胞相对于待治疗的病理物质加载校正剂，并且所述单核细胞衍生的细胞具有向上述释放的趋化因子的来源移动的性质，并且靶向存在的细胞 在所述释放的趋化因子附近，并且在诊断的情况下装载标记 呃能够检测受伤或病理部位。

公开(公告)号：US20120172415A1

公开(公告)日：2012-07-05

申请号：US13393004

申请日：2010-08-31

申请人： Thomas Voit ,  Luis Garcia ,  Valerie Robin ,  Patrick Dreyfus

发明人： Thomas Voit ,  Luis Garcia ,  Valerie Robin ,  Patrick Dreyfus

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

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2320/33 ,  C12N2310/3521

摘要： 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.

摘要翻译： 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。