IndraLab

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Cas9 increases the amount of SCN1A. 1 / 1

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"We designed a CRISPR based gene therapy for Scn1a-haplodeficient mice using multiple guide RNAs (gRNAs) in the promoter regions together with the nuclease deficient Cas9 fused to transcription activators (dCas9-VPR) to trigger the transcription of SCN1A or Scn1a in vitro."

32445790

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IndraLab

Statements

Cas9 increases the amount of SCN1A. 1 / 1

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