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Updated: Jun 9, 2025

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
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基因治疗:在治疗沙拉西米亚的革命性步骤

Jhancy Malay1, Rasha Aziz Attia Salama2, Ghania Shehzad Alam Qureshi3

  • 1Department of Pediatrics, RAK Medical and Health Sciences University, Ras Al Khaimah 11127, United Arab Emirates.

Hematology reports
|October 25, 2024
PubMed
概括

基因疗法为beta-thalassemia提供了一个有前途的,潜在的治愈治疗方法,beta-thalassemia是一种影响阿联许多人的遗传性血液疾病. 虽然创新,但必须应对高成本等挑战,以实现更广泛的采用.

关键词:
库利的贫血症是什么地中海贫血的原因大型β thalassemia 的发生.基因添加 基因添加基因编辑 基因编辑基因修饰是一种基因修饰.输血依赖的β-血症.

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