AAV‐delivered diacylglycerol kinase DGKk achieves long‐term rescue of fragile X syndrome mouse model

Abstract Fragile X syndrome (FXS) is the most frequent form of familial intellectual disability. FXS results from the lack of the RNA‐binding protein FMRP and is associated with the deregulation of signaling pathways downstream of mGluRI receptors and upstream of mRNA translation. We previously foun...

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Autors principals: Karima Habbas, Oktay Cakil, Boglárka Zámbó, Ricardos Tabet, Fabrice Riet, Doulaye Dembele, Jean‐Louis Mandel, Michaël Hocquemiller, Ralph Laufer, Françoise Piguet, Hervé Moine
Format: Artigo
Idioma:Inglês
Publicat: Wiley 2022-05-01
Col·lecció:EMBO Molecular Medicine
Matèries:
AAV
diacylglycerol kinase
Fmr1‐KO
FMRP
Fragile X syndrome
Accés en línia:https://doi.org/10.15252/emmm.202114649
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