专利检索 ap:("The Trustees of the University of Pennsylvania" OR "Ascidian Therapeutics, Inc.") AND inv:"DOOLEY, Scott, J." 第 1 页

1.

发明公开
TRANS-SPLICING MOLECULES 审中-公开

公开(公告)号：EP3781213A1

公开(公告)日：2021-02-24

申请号：EP19789043.7

申请日：2019-04-17

申请人： The Trustees of the University of Pennsylvania , Ascidian Therapeutics, Inc.

发明人： JOHNSON, Philip, R. , SCHNEPP, Bruce, C. , BENNETT, Jean , DOOLEY, Scott, J. , FISHER, Krishna, Jawaharlal , SUN, Junwei

IPC分类号： A61K48/00 , C07K14/705

2.

发明公开
COMPOSITIONS AND METHODS FOR CORRECTION OF HERITABLE OCULAR DISEASE 审中-公开

公开(公告)号：EP3377116A1

公开(公告)日：2018-09-26

申请号：EP16867296.2

申请日：2016-11-18

申请人： The Trustees of The University of Pennsylvania , Mitchell, Lloyd G.

发明人： MITCHELL, Lloyd, G. , BENNETT, Jean , BENNICELLI, Jeannette , DOOLEY, Scott, J.

IPC分类号： A61K48/00 , C07H21/02 , C07H21/04

CPC分类号： C12N15/86 , A61K48/005 , C12N15/00 , C12N2740/10043 , C12N2750/14143

摘要： A nucleic acid trans-splicing molecule is provided that can replace an exon in a targeted mammalian ocular gene carrying a defect or mutation causing an ocular disease with an exon having the naturally-occurring sequence without the defect or mutation. A method of treating an ocular disease, e.g., Stargardt's Disease, caused by a defect or mutation in a target gene, e.g., ABCA4 comprising: administering to the ocular cells of a subject having an ocular disease a composition comprising a recombinant AAV comprising a nucleic acid trans-splicing molecule as described above.