Catecholaminergic polymorphic ventricular tachycardia (CPVT) is a lethal inherited arrhythmia disorder most commonly caused by missense mutations in the RyR2 gene. The goal of this study was to determ

NIAID Data Ecosystem20

Efficient in vivo Genome Editing Prevents Hypertrophic Cardiomyopathy in Mice [Amplified_cDNA]. Efficient in vivo Genome Editing Prevents Hypertrophic Cardiomyopathy in Mice [Amplified_cDNA]

基因编辑治疗

肥厚型心肌病动物模型

Dominant pathogenic variants encoding amino acid substitutions in cardiac myosin heavy chain cause hypertrophic cardiomyopathy (HCM), a currently incurable disorder that increases risk for stroke, hea

NIAID Data Ecosystem50

Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells. Homo sapiens

基因编辑治疗

囊性纤维化基因校正

A systematic implementation of six efficiency optimizations for prime editing led to high levels of functional correction, in airway epithelial cells, of the predominant mutation that causes cystic fi

NIAID Data Ecosystem30

Therapeutic base editing of HBG promoter to reactivate high level of γ-globin expression with no detectable off-target mutations

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