US 12,419,972 B2
Muscle-specific nucleic acid regulatory elements and methods and use thereof
Thierry Vandendriessche, Bierbeek (BE); Marinee Chuah, Bierbeek (BE); and Pieter De Bleser, Buggenhout (BE)
Assigned to Vrije Universiteit Brussel, Brussels (BE); VIB VZW, Ghent (BE); and Universiteit Gent, Ghent (BE)
Filed by Vrije Universiteit Brussel, Brussels (BE); VIB VZW, Ghent (BE); and Universiteit Gent, Ghent (BE)
Filed on Jun. 9, 2021, as Appl. No. 17/343,563.
Application 17/343,563 is a continuation of application No. 15/112,795, granted, now 11,072,801, previously published as PCT/EP2015/051081, filed on Jan. 21, 2015.
Claims priority of application No. 14151960 (EP), filed on Jan. 21, 2014.
Prior Publication US 2021/0395770 A1, Dec. 23, 2021
Int. Cl. A61K 48/00 (2006.01); C12N 9/26 (2006.01); C12N 15/85 (2006.01); C12N 15/86 (2006.01); C12N 15/864 (2006.01)
CPC A61K 48/0058 (2013.01) [A61K 48/0075 (2013.01); C12N 15/85 (2013.01); C12N 15/86 (2013.01); C12N 15/8645 (2013.01); C12N 2710/10043 (2013.01); C12N 2750/14143 (2013.01); C12N 2800/22 (2013.01); C12N 2830/008 (2013.01); C12N 2830/15 (2013.01); C12N 2830/85 (2013.01)] 16 Claims
 
1. A nucleic acid expression cassette comprising at least one nucleic acid regulatory element for enhancing muscle-specific gene expression, operably linked to a promoter and a heterologous transgene, wherein said nucleic acid regulatory element comprises the sequence set forth in SEQ ID NO:1, or a functional fragment of the sequence set forth in SEQ ID NO: 1, wherein said functional fragment consists of a nucleotide sequence selected from any one of the following: positions 143 to 233 of SEQ ID NO:1, positions 240 to 310 of SEQ ID NO: 1, or positions 90 to 233 of SEQ ID NO: 1, or comprises at least 300 contiguous nucleotides from the sequence set forth in SEQ ID NO:1.