US 12,331,319 B2
Respiratory and sweat gland ionocytes
Jayaraj Rajagopal, Boston, MA (US); Aviv Regev, Cambridge, MA (US); Moshe Biton, Cambridge, MA (US); Adam Haber, Cambridge, MA (US); Daniel Montoro, Boston, MA (US); and Orit Rozenblatt-Rosen, Cambridge, MA (US)
Assigned to THE BROAD INSTITUTE, INC., Cambridge, MA (US); THE GENERAL HOSPITAL CORPORATION, Boston, MA (US); and MASSACHUSETTS INSTITUTE OF TECHNOLOGY, Cambridge, MA (US)
Appl. No. 16/604,596
Filed by The Broad Institute, Inc., Cambridge, MA (US); The General Hospital Corporation, Boston, MA (US); and Massachusetts Institute of Technology, Cambridge, MA (US)
PCT Filed Apr. 12, 2018, PCT No. PCT/US2018/027337
§ 371(c)(1), (2) Date Oct. 11, 2019,
PCT Pub. No. WO2018/191520, PCT Pub. Date Oct. 18, 2018.
Claims priority of provisional application 62/484,782, filed on Apr. 12, 2017.
Prior Publication US 2021/0115407 A1, Apr. 22, 2021
Int. Cl. C12N 5/071 (2010.01); A61K 35/42 (2015.01); A61P 11/00 (2006.01); G01N 33/50 (2006.01)
CPC C12N 5/0688 (2013.01) [A61K 35/42 (2013.01); A61P 11/00 (2018.01); G01N 33/5044 (2013.01); C12N 2501/42 (2013.01); C12N 2506/27 (2013.01)] 8 Claims
 
1. A method for increasing expression of CFTR in epithelial ionocytes, the method comprising delivering in vivo to a subject in need thereof one or more modulating agents comprising an adenovirus viral vector (AAV) comprising a polynucleotide sequence encoding FOXI1, wherein delivery of the polynucleotide sequence encoding FOXI1 increases the expression of FOXI1 and CFTR in the epithelial ionocytes.