血友病护理的范式转变:基因治疗的承诺

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Updated: Sep 11, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

Published on: August 9, 2022

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Safia Fathima Anver Pasha1, Tasnim Fathima Khalifa Anver1, Suresh Kumar Srinivasamurthy1

  • 1RAK College of Medical Sciences (RAKCOMS), RAK Medical and Health Sciences University (RAKMHSU), Ras Al Khaimah, Al Qusaidat, United Arab Emirates.

Current gene therapy
|August 11, 2025
PubMed
概括

基因疗法为血友病提供了一次性治疗,显著减少出血发作和因子替代的需要. 需要进一步的研究来确认长期的安全性和有效性.

关键词:
AAV 矢量. 在 AAV 矢量.基因治疗是一种基因疗法.血流疾病 血流疾病凝血因子是什么 凝血因子是什么埃特拉诺基因 脱离 消失 消失血友病A型 血友病A型 血友病A型血友病 B 型 血友病 B 型血液静止 血液静止 血液静止值ctocogene 罗克萨帕尔沃维克

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