Gene therapy using an ortholog of human fragile X mental retardation protein partially rescues behavioral abnormalities and EEG activity
Alexander W.M. Hooper, Hayes Wong, Yosuke Niibori, Rozita Abdoli, Subha Karumuthil‐Melethil, Chunping Qiao, Olivier Danos, Joseph T. Bruder, David R. Hampson
Abstract
KO rats. These findings support the use of AAV-based gene therapy as a treatment for FXS and specifically demonstrate the potential therapeutic benefit of human FMRP isoform 17 orthologs.
Topics & Concepts
FMR1Fragile X syndromeBiologyTransgeneGene isoformPhenotypeNeuroscienceAdeno-associated virusGenetic enhancementGeneticsGeneFragile xVector (molecular biology)Recombinant DNAGenetics and Neurodevelopmental DisordersAutism Spectrum Disorder ResearchCRISPR and Genetic Engineering