利用AXION 研究修复脆性X染色体综合症神经元

MIT Rudolf JaenischDNATet1Dnmt3aCas9 (dCas9)DNAX. Shawn Liu et al., 2016,Cell

2018CellXFragile X syndromeFXSiPSCCGG450使FMR1

dCas9Tet1CGG sgRNAFMR1CGGFMR1

X

XFragile X SyndromeFXS1/3600

X

FMR1FMRP(X)FMRPRNAFMRP使

FMR15UTRCGGXCGG200DNAFMR1FXSFMR1

Axion MaestroFXS 

WhiteheadCRISPR/dCas9Tet1iPSCCGG450使FMR12018222Cell

FXSCRISPR/Cas9CGG

FMR13CRISPR/Cas9

Axion MEAMEAiPSC3666FMR1iPSCAxion MEApostmitotic使

(MEA)WIBR1FX52mockdCas9Tet1/CGG sgRNAdCas9dTet1/CGG sgRNATet1

iPSC绿

FXSFMR1iPSC绿

LiuXiPSCGGFMR1 

John Graef,Hao WuAxion Maestro

MEA

· MEA

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