Yonghong Hong Chen

US Patent:

20210324354, Oct 21, 2021

Filed:

May 11, 2018

Appl. No.:

16/611458

Inventors:

- Philadelphia PA, US
Yonghong CHEN - Philadelphia PA, US

Assignee:

The Children's Hospital of Philadelphia - Philadelphia PA

International Classification:

C12N 9/14
A61K 45/06
C12N 7/00
A61K 48/00
A61P 25/00
C12N 15/86

Abstract:

The invention relates to sulfamidase (SGSH) and SGSH variants. SGSH and SGSH variants can be delivered by way of a recombinant adeno-associated virus (rAAV) particle to a mammal's central nervous system (CNS) to transduce CNS cells that contact cerebrospinal fluid (CSF). Target mammals for SGSH and SGSH variant administration include mammals with a deficiency or defect in SGSH expression or function.