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論文 - 一覧

  • 9 Hits
  • 検索条件 : 絞込み (MeSH = beta-Thalassemia* / genetics)
生物種 リソース名 タイトル
ヒト・動物細胞 HUDEP-2(RCB4557) Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype.
ヒト・動物細胞 HUDEP-2(RCB4557) Human cellular model systems of β-thalassemia enable in-depth analysis of disease phenotype.
ヒト・動物細胞 HUDEP-2(RCB4557) Targeting fetal hemoglobin expression to treat β hemoglobinopathies.
ヒト・動物細胞 HUDEP-2(RCB4557) Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells.
ヒト・動物細胞 HUDEP-2(RCB4557) A novel gain-of-function PIP4K2A mutation elevates the expression of β-globin and aggravates the severity of α-thalassemia.
ヒト・動物細胞 HUDEP-2(RCB4557) Prime Editor 3 Mediated Beta-Thalassemia Mutations of the HBB Gene in Human Erythroid Progenitor Cells.
ヒト・動物細胞 HUDEP-2(RCB4557) Disrupting the adult globin promoter alleviates promoter competition and reactivates fetal globin gene expression.
ヒト・動物細胞 HUDEP-2(RCB4557) Direct correction of haemoglobin E β-thalassaemia using base editors.
ヒト・動物細胞 HUDEP-2(RCB4557) Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression.