公开(公告)号：WO2010014218A3

公开(公告)日：2010-02-04

申请号：PCT/US2009/004373

申请日：2009-07-29

Applicant: ACADEMIA SINICA ,  YU, John ,  YU, Alice ,  KUO, Ming-Wei ,  CHANG, Jui-Chin

Inventor： YU, John ,  YU, Alice ,  KUO, Ming-Wei ,  CHANG, Jui-Chin

IPC: C12N15/09 ,  C12N15/63 ,  A61K48/00

Abstract: Methods and compositions for treating retinal diseases comprising therapeutic amounts of a compound selected from a normal Puf-A gene product, an active polypeptide fragment thereof, an analog thereof or a peptidomimetic thereof. Vectors, including AAV vectors comprising the therapeutic compound are provided. Puf-A compositions suitable for subretinal, intravitreal, topical, subconjunctival, retrobulbar, periocular, suprachoroidal, or intraocular administration are provided. Methods for screening siRNA, RNAi and shRNA, small molecules and monoclonal antibodies that inhibit Puf-A target activity and reduce apoptosis are provided.

公开(公告)号：WO2010014218A2

公开(公告)日：2010-02-04

申请号：PCT/US2009004373

申请日：2009-07-29

Applicant: ACADEMIA SINICA ,  YU JOHN ,  YU ALICE ,  KUO MING-WEI ,  CHANG JUI-CHIN

Inventor： YU JOHN ,  YU ALICE ,  KUO MING-WEI ,  CHANG JUI-CHIN

IPC: C12N15/09

CPC classification number: C07K14/4747 ,  A61K48/005 ,  C07K16/18 ,  C12N15/113 ,  C12N2799/025 ,  C12Q1/6876 ,  G01N33/6854

Abstract: Methods and compositions for treating retinal diseases comprising therapeutic amounts of a compound selected from a normal Puf-A gene product, an active polypeptide fragment thereof, an analog thereof or a peptidomimetic thereof. Vectors, including AAV vectors comprising the therapeutic compound are provided. Puf-A compositions suitable for subretinal, intravitreal, topical, subconjunctival, retrobulbar, periocular, suprachoroidal, or intraocular administration are provided. Methods for screening siRNA, RNAi and shRNA, small molecules and monoclonal antibodies that inhibit Puf-A target activity and reduce apoptosis are provided.

Abstract translation: 用于治疗视网膜疾病的方法和组合物包括治疗量的选自正常Puf-A基因产物，其活性多肽片段，其类似物或肽模拟物的化合物。 提供载体，包括包含治疗化合物的AAV载体。 提供适用于视网膜下，玻璃体内，局部，结膜下结膜，球后，眼周，脉络膜或眼内给药的Puf-A组合物。 提供了筛选siRNA，RNAi和shRNA，抑制Puf-A靶活性和减少细胞凋亡的小分子和单克隆抗体的方法。