US 12,403,204 B2
Gene therapy for ocular disorders
Jean Bennett, Bryn Mawr, PA (US); Jeannette Bennicelli, Philadelphia, PA (US); and Junwei Sun, Philadelphia, PA (US)
Assigned to The Trustees of the University of Pennsylvania, Philadelphia, PA (US)
Filed by The Trustees of the University of Pennsylvania, Philadelphia, PA (US)
Filed on Jul. 7, 2021, as Appl. No. 17/369,525.
Application 17/369,525 is a division of application No. 16/061,530, granted, now 11,090,392, previously published as PCT/US2016/066402, filed on Dec. 13, 2016.
Claims priority of provisional application 62/266,789, filed on Dec. 14, 2015.
Prior Publication US 2021/0330816 A1, Oct. 28, 2021
Int. Cl. A61K 48/00 (2006.01); A61K 9/00 (2006.01); C07K 14/47 (2006.01); C12N 15/86 (2006.01)
CPC A61K 48/0058 (2013.01) [A61K 9/0048 (2013.01); C07K 14/47 (2013.01); C12N 15/86 (2013.01); C12N 2750/14143 (2013.01); C12N 2799/022 (2013.01); C12N 2799/04 (2013.01); C12N 2800/107 (2013.01); C12N 2800/22 (2013.01); C12N 2840/002 (2013.01); C12N 2840/007 (2013.01)] 10 Claims
 
1. A method for treating achromatopsia in a human subject in need thereof, said method comprising administering to the human subject subretinally an AAV vector comprising an AAV capsid and a nucleic acid sequence packaged therein, wherein the nucleic acid sequence comprises i) AAV ITR sequences, ii) a nucleic acid sequence encoding human cyclic nucleotide gated channel alpha 3 (CNGA3), and iii) expression control sequences that direct expression of the CNGA3 in a host cell, wherein the nucleic acid sequence encoding the human CNGA3 sequence comprises SEQ ID NO: 9 or SEQ ID NO: 11.