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Gene Therapy for Cystic Fibrosis Paved the Way for the Use of Adeno-Associated Virus in Gene Therapy.
Human Gene Therapy ( IF 4.2 ) Pub Date : 2020-05-08 , DOI: 10.1089/hum.2020.046
William B Guggino 1 , Liudmila Cebotaru 2
Affiliation  

Shortly after the cystic fibrosis (CF) gene was identified in 1989, the race began to develop a gene therapy for this condition. Major efforts utilized full-length cystic fibrosis transmembrane conductance regulator packaged into adenovirus, adeno-associated virus (AAV), or liposomes and delivered to the airways. The drive to find a treatment for CF based on gene therapy drove the early stages of gene therapy in general, particularly those involving AAV gene therapy. Since general overviews of CF gene therapy have already been published, this review considers specifically the efforts using AAV and is focused on honoring the contributions of Dr. Barrie Carter.

中文翻译:

囊性纤维化的基因治疗为腺相关病毒在基因治疗中的应用铺平了道路。

在 1989 年发现囊性纤维化 (CF) 基因后不久,种族就开始开发针对这种情况的基因疗法。主要的努力是利用包装成腺病毒、腺相关病毒 (AAV) 或脂质体的全长囊性纤维化跨膜电导调节剂并输送到气道。寻找基于基因治疗的 CF 治疗方法的动力推动了基因治疗的早期阶段,尤其是那些涉及 AAV 基因治疗的阶段。由于 CF 基因治疗的总体概述已经发表,因此本综述特别考虑了使用 AAV 的努力,并重点表彰 Barrie Carter 博士的贡献。
更新日期:2020-05-08
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