公开(公告)号：US20140294771A1

公开(公告)日：2014-10-02

申请号：US14113205

申请日：2012-04-20

申请人： David V. Schaffer ,  Ryan R. Klimczak ,  James T. Koerber ,  John G. Flannery ,  Deniz Dalkara Mourot ,  Meike Visel ,  Leah C.T. Byrne

发明人： David V. Schaffer ,  Ryan R. Klimczak ,  James T. Koerber ,  John G. Flannery ,  Deniz Dalkara Mourot ,  Meike Visel ,  Leah C.T. Byrne

IPC分类号： C12N15/86 ,  C07K14/005

CPC分类号： C12Q1/701 ,  A61K9/0019 ,  A61K9/0048 ,  A61K38/1709 ,  A61K48/0008 ,  A61K48/0025 ,  A61K48/0075 ,  C07K14/005 ,  C07K2319/33 ,  C12N7/00 ,  C12N15/113 ,  C12N15/115 ,  C12N15/86 ,  C12N2310/14 ,  C12N2310/16 ,  C12N2320/32 ,  C12N2750/14021 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14142 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14152 ,  C12N2810/40 ,  C12Q1/70 ,  C12Q2600/158 ,  C12Q2600/16 ,  G01N33/5008 ,  G01N33/5058

摘要： The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells, when administered via intravitreal injection, compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.