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CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease.

Akshay SharmaJaap-Jan BoelensMaria CancioJane S HankinsPrafulla BhadMarjohn AzizyAndrew LewandowskiXiaojun ZhaoShripad ChitnisRadhika PeddintiYan ZhengNeena KapoorFabio CiceriTimothy MaclachlanYi YangYi LiuJianping YuanUlrike NaumannVionnie W C YuSusan C StevensonSerena De VitaJames L LaBelle
Published in: The New England journal of medicine (2023)
gene promoters was an effective strategy for induction of fetal hemoglobin. Infusion of autologous OTQ923 into three participants with severe sickle cell disease resulted in sustained induction of red-cell fetal hemoglobin and clinical improvement in disease severity. (Funded by Novartis Pharmaceuticals; ClinicalTrials.gov number, NCT04443907.).
Keyphrases
  • sickle cell disease
  • crispr cas
  • genome editing
  • cell therapy
  • single cell
  • red blood cell
  • low dose
  • bone marrow
  • copy number
  • genome wide
  • stem cells
  • gene expression
  • transcription factor
  • genome wide identification