Target intelligence / Profile preview

Human immunodeficiency virus type 1 proviral DNA at tat exons (HIV-1 proviral DNA (tat))

Target
HIV-1 proviral DNA (tat)
Molecular classification
Nucleic acid, Viral DNA, Proviral DNA
01

Overview

Human immunodeficiency virus type 1 (HIV-1) proviral DNA at the tat exons represents a critical genomic target for curative strategies against HIV/AIDS. The tat gene encodes the Trans-Activator of Transcription (Tat) protein, which is essential for the efficient elongation of viral transcripts by binding to the Trans-Activation Response (TAR) element. Without a functional Tat protein, HIV-1 transcription remains at basal, inefficient levels, effectively preventing viral replication and the production of new virions. Targeting the DNA sequence of the tat exons, particularly through gene-editing technologies like CRISPR/Cas9, aims to permanently disrupt or excise the proviral genome from host cells. This approach is a cornerstone of 'excision' therapy, which seeks to eliminate the latent HIV reservoir that persists in resting CD4+ T cells despite antiretroviral therapy (ART). By disabling the tat gene at the DNA level, researchers hope to achieve a functional cure where the virus can no longer rebound even in the absence of daily medication.

Other names
HIV-1 tat geneHIV-1 trans-activator of transcription DNAHIV-1 proviral tat exonsTat exon 1Tat exon 2
02

Mechanism of action

Gene editing and excision of proviral DNA sequences to disrupt viral replication and eliminate the latent reservoir.

03

Biological functions

Viral replicationTranscription regulationViral gene expressionLatency maintenance
04

Disease associations

InfectionAcquired immunodeficiency syndrome (AIDS)
05

Safety considerations

Off-target DNA cleavageIncomplete excision of provirusPotential for viral escape mutationsImmunogenicity of delivery vectors (e.g., AAV)Integration of CRISPR components into host genome
06

Interacting drugs

EBT-101

1 more in the full profile.

07

Biomarkers

HIV-1 DNA levelsPlasma viral load (HIV-1 RNA)CD4+ T-cell countCell-associated HIV-1 RNA

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