Gene therapy using an ortholog of human fragile X mental retardation protein partially rescues behavioral abnormalities and EEG activity

Gene therapy using an ortholog of human fragile X mental retardation protein partially rescues behavioral abnormalities and EEG activity

Fragile X syndrome (FXS), a neurodevelopmental disorder with no known cure, is caused by a lack of expression of the fragile X mental retardation protein (FMRP). As a single-gene disorder, FXS is an excellent candidate for viral-vector-based gene therapy, although that is complicated by the existenc...

Full description

Bibliographic Details
Main Authors: Alexander W.M. Hooper, Hayes Wong, Yosuke Niibori, Rozita Abdoli, Subha Karumuthil-Melethil, Chunping Qiao, Olivier Danos, Joseph T. Bruder, David R. Hampson
Format: Article
Language:English
Published: Elsevier 2021-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
AAV
autism
cerebrospinal fluid
EEG
epilepsy
GABA
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121001157

Internet

http://www.sciencedirect.com/science/article/pii/S2329050121001157

Similar Items