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

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Autor(es):
Domingues, Wilson ; Folgosi, Victor angelo ; Sanabani, Sabri Saeed ; Leite Junior, Pedro Domingos ; Assone, Tatiane ; Casseb, Jorge
Número total de Autores: 6
Tipo de documento: Artigo Científico
Fonte: Revista do Instituto de Medicina Tropical de São Paulo; v. 66, p. 9-pg., 2024-01-01.
Resumo

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. (AU)

Processo FAPESP: 18/07239-2 - Incidência, fatores de risco, mecanismos patogênicos e prevenção de distúrbios neurocognitivos (HAND) entre indivíduos infectados com HIV-1
Beneficiário:Jorge Simao do Rosario Casseb
Modalidade de apoio: Auxílio à Pesquisa - Temático
Processo FAPESP: 22/00132-3 - Incidência, fatores de risco, e mecanismos patogênicos nos distúrbios neurocognitivos associados ao HIV-1 (HAND)
Beneficiário:Wilson Domingues
Modalidade de apoio: Bolsas no Brasil - Pós-Doutorado
Processo FAPESP: 23/14320-9 - Estratégias de tratamento para infecção pelo HTLV-1: edição genética e modulação imunológica em células de primatas não humanos
Beneficiário:Víctor Ângelo Folgosi
Modalidade de apoio: Bolsas no Brasil - Doutorado