US 12,121,567 B2
Methods to treat mucopolysaccharidosis type II or deficiency in iduronate-2-sulfatase using a recombinant adeno-associated virus (AAV) vector encoding iduronate-2-sulfatase
R. Scott McIvor, St. Louis Park, MN (US); Lalitha R. Belur, St. Paul, MN (US); Walter Low, Shorewood, MN (US); Carolyn Fairbanks, St. Paul, MN (US); and Karen Kozarsky, Bala Cynwyd, PA (US)
Assigned to Regents of the University of Minnesota, Minneapolis, MN (US); and REGENXBIO Inc., Rockville, MD (US)
Filed by Regents of the University of Minnesota, Minneapolis, MN (US); and REGENXBIO Inc., Rockville, MD (US)
Filed on Sep. 27, 2017, as Appl. No. 15/717,358.
Application 15/717,358 is a division of application No. 14/889,750, granted, now 9,827,295, previously published as PCT/US2014/038209, filed on May 15, 2014.
Claims priority of provisional application 61/823,757, filed on May 15, 2013.
Prior Publication US 2018/0099030 A1, Apr. 12, 2018
Int. Cl. C12N 15/11 (2006.01); A61K 38/43 (2006.01); A61K 38/47 (2006.01); A61K 48/00 (2006.01); C12N 7/00 (2006.01); C12N 15/00 (2006.01); C12N 15/113 (2010.01); C12N 15/52 (2006.01); C12N 15/85 (2006.01); C12N 15/86 (2006.01); C12N 15/861 (2006.01)
CPC A61K 38/47 (2013.01) [C12N 7/00 (2013.01); C12N 15/86 (2013.01); A61K 48/00 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14171 (2013.01); C12N 2830/007 (2013.01); C12Y 302/01076 (2013.01)] 25 Claims
 
1. A method to treat one or more symptoms of mucopolysaccharidosis type II (MPSII) in a human having MPSII, consisting of: administering to a cisterna magna of the human a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 or rAAVrh10 vector comprising an open reading frame encoding iduronate-2-sulfatase in a physiologically compatible buffer effective to treat the one or more symptoms of MPSII.