US 12,257,321 B2
Optimized mini-dystrophin genes and expression cassettes and their use
Xiao Xiao, Chapel Hill, NC (US); Juan Li, Chapel Hill, NC (US); Chunping Qiao, Chapel Hill, NC (US); Scott W. J. McPhee, Chapel Hill, NC (US); Richard J. Samulski, Hillsborough, NC (US); and Maritza McIntyre, Rockville, MD (US)
Assigned to The University of North Carolina at Chapel Hill, Chapel Hill, NC (US); and Bamboo Therapeutics, Inc., Chapel Hill, NC (US)
Filed by The University of North Carolina at Chapel Hill, Chapel Hill, NC (US); and Bamboo Therapeutics, Inc., Chapel Hill, NC (US)
Filed on Jan. 5, 2023, as Appl. No. 18/150,377.
Application 18/150,377 is a continuation of application No. 16/787,938, filed on Feb. 11, 2020, granted, now 11,547,765.
Application 16/787,938 is a continuation of application No. 15/628,268, filed on Jun. 20, 2017, abandoned.
Claims priority of provisional application 62/516,449, filed on Jun. 7, 2017.
Claims priority of provisional application 62/352,675, filed on Jun. 21, 2016.
Prior Publication US 2023/0398236 A1, Dec. 14, 2023
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 48/00 (2006.01); A61K 38/17 (2006.01); A61K 45/06 (2006.01); C07K 14/47 (2006.01); C07K 14/78 (2006.01); C12N 15/86 (2006.01)
CPC A61K 48/0058 (2013.01) [A61K 38/1709 (2013.01); A61K 45/06 (2013.01); C07K 14/4708 (2013.01); C07K 14/78 (2013.01); C12N 15/86 (2013.01); A01K 2217/075 (2013.01); A01K 2227/10 (2013.01); A01K 2227/105 (2013.01); A01K 2267/0306 (2013.01); C12N 2750/14143 (2013.01); C12N 2800/22 (2013.01); C12N 2830/008 (2013.01)] 14 Claims
 
1. A recombinant AAV (rAAV) particle comprising an AAV9 capsid and a vector genome, said genome comprising a first AAV2 inverted terminal repeat (ITR), a muscle-specific transcriptional regulatory element operably linked to a nucleotide sequence encoding a human mini-dystrophin protein consisting of the amino acid sequence of SEQ ID NO: 7, a transcription termination sequence, and a second AAV2 ITR.