Tech Comm Available Technologies :: The Research Institute at Nationwide Children's Hospital, Columbus, Ohio

Details

Project TitleRecombinant Adeno-Associated Virus Vector Gene Therapy for MPS II
Track Code2016-001
Short Description

Researchers at Nationwide Children's Hospital are developing a gene therapy vector (systemic delivery) to cross the BBB for treatment of the CNS and also for somatic manifestation of MPS.

Abstract

Hunter's syndrome, or mucopolysaccharidosis II (MPS II), is a lysosomal storage disease caused by a deficiency of enzyme iduronate-2-sulfatase (I2S). The greatest challenge in developing therapies for MPS IIis to achieve efficient central nervous system (CNS) delivery across the blood-brain barrier (BBB). Researchers at Nationwide Children's Hospital are developing a gene therapy vector (systemic delivery) to cross the BBB for treatment of the CNS and also for somatic manifestation of MPS.

 
TagsHunter's syndrome, mucopolysaccharidosis II, MPS II, lysosomal storage disase, enzyme iduronate-2-sultatase, I2S, cns, bbb, gene therapy, vector, systemic delivery
 
Posted DateSep 25, 2017 3:14 PM

Researcher

Name
Douglas McCarty
Haiyan Fu

Manager

Name
Margaret Barkett

Inquiry

Intellectual Property

Patent Number Issue Date Type Country of Filing
None None Other Patent None
Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000