公开(公告)号：EP2970972B1

公开(公告)日：2018-08-15

申请号：EP14709644.0

申请日：2014-03-11

申请人： Fondazione Telethon

发明人： SURACE, Enrico Maria ,  BANFI, Sandro ,  CONTE, Ivan ,  KARALI, Marianthi ,  MARROCCO, Elena

IPC分类号： C12N15/113 ,  A61K31/7105 ,  A61P27/02

CPC分类号： A61K48/005 ,  A61K31/7105 ,  C12N15/113 ,  C12N15/86 ,  C12N2310/141 ,  C12N2710/10032

摘要： The present invention relates to at least one agent capable of increasing the level of one or more miRNA in a cell or cells of a subject, said miRNA comprising the sequence UUCCCUU, for use in the treatment and/or prevention of a retinal dystrophy, in particular characterized by photoreceptor degeneration, relative pharmaceutical compositions, nucleic acids, vectors and host cells.

公开(公告)号：EP2970972A1

公开(公告)日：2016-01-20

申请号：EP14709644.0

申请日：2014-03-11

申请人： Fondazione Telethon

发明人： SURACE, Enrico Maria ,  BANFI, Sandro ,  CONTE, Ivan ,  KARALI, Marianthi ,  MARROCCO, Elena

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61P27/02

CPC分类号： A61K48/005 ,  A61K31/7105 ,  C12N15/113 ,  C12N15/86 ,  C12N2310/141 ,  C12N2710/10032

摘要： The present invention relates to at least one agent capable of increasing the level of one or more miRNA in a cell or cells of a subject, said miRNA comprising the sequence UUCCCUU, for use in the treatment and/or prevention of a retinal dystrophy, in particular characterized by photoreceptor degeneration, relative pharmaceutical compositions, nucleic acids, vectors and host cells.

摘要翻译： 本发明涉及能够增加受试者的细胞或细胞中的一种或多种miRNA的水平的至少一种试剂，所述miRNA包含用于治疗和/或预防视网膜营养不良的序列UUCCCUU，其用于治疗和/或预防视网膜营养不良症 其特征在于感光器变性，相对药物组合物，核酸，载体和宿主细胞。

公开(公告)号：EP3071592A2

公开(公告)日：2016-09-28

申请号：EP14802414.4

申请日：2014-11-20

申请人： Fondazione Telethon

发明人： SURACE, Enrico Maria ,  MARROCCO, Elena ,  BOTTA, Salvatore

IPC分类号： C07K14/47 ,  A61K48/00 ,  C12N15/86 ,  C12Q1/68

CPC分类号： C07K14/4703 ,  A61K38/00 ,  A61K48/00 ,  A61K48/005 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2830/008

摘要： The present invention relates to proteins consisting of an artificial DNA-binding domain (DBD) and related molecules and uses thereof. In particular, the proteins are ZF-DBD or TALE-DBD and are used for the treatment of eye disorders caused by gain of function mutation. The disorder may be ADRP, in particular ADRP caused by mutation in the rhodopsin gene. The present invention also relates to a method to identify cis-regulatory elements and to modulate them via DBDs.