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HIV Gene Therapy: An Update
Human Gene Therapy ( IF 3.9 ) Pub Date : 2021-01-18 , DOI: 10.1089/hum.2020.159
Tatjana I Cornu 1, 2, 3 , Claudio Mussolino 1, 2, 3 , Matthias C Müller 3, 4, 5 , Claudia Wehr 2, 3, 6 , Winfried V Kern 3, 4 , Toni Cathomen 1, 2, 3
Affiliation  

Progress in antiretroviral therapy has considerably reduced mortality and notably improved the quality of life of individuals infected with HIV since the pandemic began some 40 years ago. However, drug resistance, treatment-associated toxicity, adherence to medication, and the need for lifelong therapy have remained major challenges. While the development of an HIV vaccine has remained elusive, considerable progress in developing innovative cell and gene therapies to treat HIV infection has been made. This includes immune cell therapies, such as chimeric antigen receptor T cells to target HIV infected cells, as well as gene therapies and genome editing strategies to render the patient's immune system resistant to HIV. Nonetheless, all of these attempts to achieve a functional cure in HIV patients have failed thus far. This review introduces the clinical as well as the technical challenges of treating HIV infection, and summarizes the most promising cell and gene therapy concepts that have aspired to bring about functional cure for people living with HIV. It further discusses socioeconomic aspects as well as future directions for developing cell and gene therapies with a potential to be an effective one-time treatment with minimal toxicity.

中文翻译:

HIV基因治疗:更新

自大约 40 年前大流行开始以来,抗逆转录病毒疗法的进展已大大降低了艾滋病毒感染者的死亡率,并显着提高了感染者的生活质量。然而,耐药性、治疗相关毒性、药物依从性和终身治疗的需求仍然是主要挑战。虽然 HIV 疫苗的开发仍然难以捉摸,但在开发用于治疗 HIV 感染的创新细胞和基因疗法方面已经取得了相当大的进展。这包括免疫细胞疗法,例如靶向 HIV 感染细胞的嵌合抗原受体 T 细胞,以及基因疗法和基因组编辑策略,以使患者的免疫系统对 HIV 产生抵抗力。尽管如此,迄今为止,所有这些在 HIV 患者中实现功能性治愈的尝试都失败了。这篇综述介绍了治疗 HIV 感染的临床和技术挑战,并总结了最有希望为 HIV 感染者带来功能性治愈的细胞和基因治疗概念。它进一步讨论了社会经济方面以及开发细胞和基因疗法的未来方向,这些疗法有可能成为一种有效的一次性治疗,毒性最小。
更新日期:2021-01-21
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