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...

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Detalles Bibliográficos
Autores principales: Hooper, Alexander W.M., Wong, Hayes, Niibori, Yosuke, Abdoli, Rozita, Karumuthil-Melethil, Subha, Qiao, Chunping, Danos, Olivier, Bruder, Joseph T., Hampson, David R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8399347/
https://www.ncbi.nlm.nih.gov/pubmed/34485605
http://dx.doi.org/10.1016/j.omtm.2021.06.013

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8399347/
https://www.ncbi.nlm.nih.gov/pubmed/34485605
http://dx.doi.org/10.1016/j.omtm.2021.06.013

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