公开(公告)号：US20170007720A1

公开(公告)日：2017-01-12

申请号：US15120425

申请日：2015-02-18

Applicant: UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC.

Inventor： Shannon E. Boye ,  Frank Dyka ,  Sanford L. Boye ,  William W. Hauswirth

IPC: A61K48/00 ,  C12N15/86 ,  C12N7/00 ,  A61K45/06 ,  A61K38/17

CPC classification number: A61K48/0058 ,  A61K38/1709 ,  A61K45/06 ,  A61K48/0075 ,  A61K48/0083 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14123 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14171 ,  C12N2830/008

Abstract: Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations that include them. Also disclosed are methods of preparing and using novel capsid-protein-mutated rAAV vector constructs in a variety of diagnostic and therapeutic applications including, inter alia, as delivery agents for diagnosis, treatment, or amelioration of one or more diseases, disorders, or dysfunctions of the mammalian eye. Also disclosed are methods for intravitreal delivery of therapeutic gene constructs to retinal neuron cells, and specifically to ON bipolar cells, of the mammalian eye, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications including the treatment of retinitis pigmentosa, melanoma-associated retinopathy, and congenital stationary night blindness.

Abstract translation: 公开了衣壳修饰的rAAV表达载体，以及感染性病毒粒子，组合物和包括它们的药物制剂。 还公开了在各种诊断和治疗应用中制备和使用新的衣壳蛋白突变的rAAV载体构建体的方法，包括特别地，作为用于诊断，治疗或改善一种或多种疾病，障碍或功能障碍的递送剂 的哺乳动物眼睛。 还公开了将治疗性基因构建体玻璃体内递送给视网膜神经元细胞，特别是哺乳动物眼睛的ON双极细胞的方法，以及所公开的组合物在制备多种体外和/或 体内应用，包括治疗色素性视网膜炎，黑素瘤相关性视网膜病变和先天性静止性夜盲。