[基因治疗:从技术到现实]。

Harefuah Pub Date : 2024-02-01
Eithan Galun
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引用次数: 0

摘要

简介基因疗法在过去几十年中取得了重大成就。这些成就涉及众多医学领域,包括代谢、肿瘤、感染和再生。如今,美国和欧洲的监管机构已批准多种基因疗法用于临床。然而,我们仍然面临着许多重大障碍,包括1.最近我们了解到,许多基因治疗方法都非常昂贵。在 COVID-19 大流行期间,基因疫苗接种证明了它的有效性,其中合成递送系统中的 RNA 非常有效。本综述将重点介绍基因疗法中三种快速发展的技术:1.CAR-T 细胞;2.CRISPR-Cas;3.RNAi。RNAi。
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[GENE THERAPY: FROM TECHNOLOGY TO REALITY].

Introduction: Gene therapy has made major achievements in the last few decades. These were in numerous medical disciplines, including metabolic, oncologic, infectious and regenerative. As of today, regulatory agencies, both in the USA and Europe, approved for clinical usage numerous gene therapy treatments. However, we are still facing a number of significant obstacles including: 1. Efficient delivery systems, 2. Immunological responses, and 3. recently we have learned that many gene therapy approaches are very expensive. The COVID-19 pandemic was a period in which genetic vaccination had proved its efficacy, by which RNA in a synthetic delivery system was very effective. This review will focus on three fast developing technologies in gene therapy: 1. CAR-T cells, 2. CRISPR-Cas and 3. RNAi.

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