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

Authors

  • Wilson Domingues Universidade de São Paulo, Faculdade de Medicina, Divisão de Dermatologia, Laboratório de Investigação Médica LIM-56, São Paulo, São Paulo, Brazil https://orcid.org/0000-0003-1045-8181
  • Victor Ângelo Folgosi Universidade de São Paulo, Faculdade de Medicina, Divisão de Dermatologia, Laboratório de Investigação Médica LIM-56, São Paulo, São Paulo, Brazil https://orcid.org/0000-0002-0114-7587
  • Sabri Saeed Sanabani Universidade de São Paulo, Faculdade de Medicina, Divisão de Dermatologia, Laboratório de Investigação Médica LIM-56, São Paulo, São Paulo, Brazil https://orcid.org/0000-0002-8876-8262
  • Pedro Domingos Leite Junior Universidade de São Paulo, Faculdade de Medicina, Divisão de Dermatologia, Laboratório de Investigação Médica LIM-56, São Paulo, São Paulo, Brazil https://orcid.org/0000-0002-0326-0487
  • Tatiane Assone Universidade de São Paulo, Faculdade de Medicina, Divisão de Dermatologia, Laboratório de Investigação Médica LIM-56, São Paulo, São Paulo, Brazil https://orcid.org/0000-0003-0993-4523
  • Jorge Casseb Universidade de São Paulo, Faculdade de Medicina, Divisão de Dermatologia, Laboratório de Investigação Médica LIM-56, São Paulo, São Paulo, Brazil https://orcid.org/0000-0002-4553-2559

DOI:

https://doi.org/10.1590/

Keywords:

HTLV-1, Gene editing, CRISPR-Cas9., TAX, HBZ

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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Published

2024-09-27

Issue

Section

Review

Funding data

How to Cite

Domingues, W., Folgosi, V. Ângelo, Sanabani, S. S., Leite Junior, P. D., Assone, T., & Casseb, J. (2024). Novel approaches for HTLV-1 therapy: innovative applications of CRISPR-Cas9. Revista Do Instituto De Medicina Tropical De São Paulo, 66, e48. https://doi.org/10.1590/