	US 11,717,579 B2
	Globin gene therapy for treating hemoglobinopathies
Michel Sadelain, New York, NY (US); Isabelle Riviere, New York, NY (US); Jorge Mansilla-Soto, New York, NY (US); Xiuyan Wang, New York, NY (US); George Stamatoyannopoulos, Seattle, WA (US); John Stamatoyannopoulos, Seattle, WA (US); and Mingdong Liu, Seattle, WA (US)
Assigned to MEMORIAL SLOAN-KETTERING CANCER CENTER, New York, NY (US); and UNIVERSITY OF WASHINGTON, Seattle, WA (US)
Filed by MEMORIAL SLOAN-KETTERING CANCER CENTER, New York, NY (US); and UNIVERSITY OF WASHINGTON, Seattle, WA (US)
Filed on Mar. 3, 2017, as Appl. No. 15/449,416.
Application 15/449,416 is a continuation of application No. PCT/US2015/048698, filed on Sep. 4, 2015.
Claims priority of provisional application 62/045,997, filed on Sep. 4, 2014.
Prior Publication US 2017/0173185 A1, Jun. 22, 2017
Int. Cl. C12N 15/86 (2006.01); C12N 15/63 (2006.01); A61K 48/00 (2006.01); C12N 15/11 (2006.01); C07K 14/805 (2006.01); C12N 9/22 (2006.01); C12N 15/85 (2006.01)

CPC A61K 48/0066 (2013.01) [A61K 48/0058 (2013.01); C07K 14/805 (2013.01); C12N 9/22 (2013.01); C12N 15/85 (2013.01); C12N 15/86 (2013.01); C12N 2740/10043 (2013.01); C12N 2740/15043 (2013.01); C12N 2740/16043 (2013.01); C12N 2830/008 (2013.01); C12N 2830/15 (2013.01); C12N 2830/30 (2013.01); C12N 2830/40 (2013.01); C12N 2830/46 (2013.01); C12N 2830/48 (2013.01); C12N 2830/50 (2013.01)]

61 Claims

1. A viral vector comprising an expression cassette comprising an insulator that comprises the nucleotide sequence set forth in SEQ ID NO:1, SEQ ID NO: 24, or SEQ ID NO: 25, and a globin gene operably linked to a β-globin locus control region (LCR) region.