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Updated: Jun 23, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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为了治愈钻石黑粉无血症:对基因治疗的看法

Matilde Vale1, Jan Prochazka1,2, Radislav Sedlacek1,2

  • 1Laboratory of Transgenic Models of Diseases, Institute of Molecular Genetics of the Czech Academy of Sciences, v.v.i, 252 50 Vestec, Czech Republic.

Cells
|June 19, 2024
PubMed
概括

钻石-布莱克芬贫血 (DBA) 是一种罕见的遗传疾病,在基因疗法方面表现有前途. 隐形病毒载体和CRISPR/Cas9为这种核糖体病变提供了潜在的治愈解决方案.

关键词:
这就是CRISPR/Cas9的作用.钻石黑粉丝贫血症 钻石黑粉丝贫血症基因治疗的基因疗法造血干细胞移植 造血干细胞移植这是一个lentiviral向量.不整合的晶状病毒载体.罕见的遗传性疾病 罕见的遗传性疾病核糖体蛋白质基因 核糖体蛋白质基因核糖体变异症是一种核糖体变异症.

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