	US 11,891,616 B2
	Transgene cassettes designed to express a human MECP2 gene
Steven J. Gray, Southlake, TX (US); and Sarah Sinnett, Farmers Branch, TX (US)
Assigned to Board of Regents of the University of Texas System, Austin, TX (US)
Filed by THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM, Austin, TX (US)
Filed on Dec. 4, 2020, as Appl. No. 17/112,299.
Claims priority of provisional application 63/047,596, filed on Jul. 2, 2020.
Claims priority of provisional application 63/008,159, filed on Apr. 10, 2020.
Claims priority of provisional application 62/946,696, filed on Dec. 11, 2019.
Claims priority of provisional application 62/944,209, filed on Dec. 5, 2019.
Prior Publication US 2021/0180085 A1, Jun. 17, 2021
Int. Cl. C12N 15/86 (2006.01); C12N 15/113 (2010.01)

CPC C12N 15/86 (2013.01) [C12N 15/113 (2013.01); C12N 2310/141 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01)]

31 Claims

1. An rAAV vector, comprising, in a 5′ to 3′ direction:

a) a first AAV ITR sequence;

b) a promoter sequence;

c) a transgene nucleic acid molecule, wherein the transgene nucleic acid molecule comprises a nucleic acid sequence encoding for an MeCP2-derived polypeptide,

wherein the MeCP2-derived polypeptide is a miniMeCP2 polypeptide;

d) a regulatory sequence comprising a sequence with at least 80% identity to the nucleic acid sequence set forth in SEQ ID NO:13; and

e) a second AAV ITR sequence.