Novel approaches for HTLV-1 therapy: innovative applications of CRISPR-Cas9.

IF 1.5 4区 医学 Q4 INFECTIOUS DISEASES Revista Do Instituto De Medicina Tropical De Sao Paulo Pub Date : 2024-08-26 eCollection Date: 2024-01-01 DOI:10.1590/S1678-9946202466048
Wilson Domingues, Victor Ângelo Folgosi, Sabri Saeed Sanabani, Pedro Domingos Leite Junior, Tatiane Assone, Jorge Casseb
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Abstract

The human T-cell lymphotropic virus type 1 (HTLV-1) is a single-stranded positive-sense RNA virus that belongs to the Retroviridae family, genus Deltaretro, and infects approximately five to 10 million people worldwide. Although a significant number of individuals living with HTLV-1 remain asymptomatic throughout their lives, some develop one or more severe clinical conditions, such as HTLV-1-associated myelopathy/tropical spastic paraparesis (HAM/TSP), a progressive and debilitating disease, and/or a subtype of non-Hodgkin's lymphoma with a more threatening course known as adult T-cell leukemia/lymphoma (ATLL). Moreover, current therapeutic options are limited and focus primarily on treating symptoms and controlling viral latency. CRISPR-Cas9 gene editing is proposed as a promising tool to address the intricate links associated with HTLV-1. By targeting or silencing key genes during initial infection and dysregulating immune signaling pathways, CRISPR-Cas9 offers potential intervention opportunities. In this review, we address the therapeutic potential of CRISPR-Cas9 gene editing, as well as examine the primary mechanisms involved in editing potential target genes and discuss the existing evidence in the current scientific literature.

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治疗 HTLV-1 的新方法:CRISPR-Cas9 的创新应用。
人类 T 细胞淋巴细胞病毒 1 型(HTLV-1)是一种单链正义 RNA 病毒,属于逆转录病毒科 Deltaretro 属,全球约有 500 万到 1000 万人感染该病毒。虽然相当多的 HTLV-1 感染者终生无症状,但有些人却会出现一种或多种严重的临床症状,如 HTLV-1 相关性脊髓病/热带痉挛性截瘫(HAM/TSP)(一种进行性衰弱性疾病)和/或非霍奇金淋巴瘤的一种亚型,即成人 T 细胞白血病/淋巴瘤(ATLL),其病程更具威胁性。此外,目前的治疗方案有限,主要集中在治疗症状和控制病毒潜伏期。CRISPR-Cas9基因编辑被认为是解决与HTLV-1相关的错综复杂联系的一种有前途的工具。通过靶向或沉默初始感染期间的关键基因以及失调的免疫信号通路,CRISPR-Cas9 提供了潜在的干预机会。在这篇综述中,我们将探讨 CRISPR-Cas9 基因编辑的治疗潜力,研究编辑潜在靶基因所涉及的主要机制,并讨论当前科学文献中的现有证据。
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来源期刊
CiteScore
3.60
自引率
5.30%
发文量
100
审稿时长
6-12 weeks
期刊介绍: The Revista do Instituto de Medicina Tropical de São Paulo (Journal of the São Paulo Institute of Tropical Medicine) is a journal devoted to research on different aspects of tropical infectious diseases. The journal welcomes original work on all infectious diseases, provided that data and results are directly linked to human health. The journal publishes, besides original articles, review articles, case reports, brief communications, and letters to the editor. The journal publishes manuscripts only in English. From 2016 on, the Revista do Instituto de Medicina Tropical de São Paulo (Journal of the São Paulo Institute of Tropical Medicine) is published online only, maintaining the free access. For more information visit: - http://www.scielo.br/rimtsp - http://www.imt.usp.br/revista-imt/
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