CRISPR/Cas9 screen in human iPSC-derived cortical neurons identifies NEK6 as a novel disease modifier of C9orf72 poly(PR) toxicity.
Wenting GuoHaibo WangArun Kumar TharkeshwarJulien CouthouisElke BraemsPegah MasroriEvelien Van SchoorYannan FanKaran AhujaMatthieu MoisseMaarten JacquemynRodrigo Furtado Madeiro da CostaMadhavsai GajjarSriram BalusuTine TricotLaura FumagalliNicole HersmusRekin's JankyFrancis ImpensPieter Vanden BergheRitchie HoDietmar Rudolf ThalRik VandenbergheMuralidhar L HegdeSiddharthan ChandranBart De StrooperDirk DaelemansPhilip Van DammeLudo Van Den BoschCatherine VerfailliePublished in: Alzheimer's & dementia : the journal of the Alzheimer's Association (2022)
We identified NEK6, which regulates poly(PR)-mediated p53-related DNA damage, as a novel therapeutic target for C9orf72 FTD/ALS.