Journal of neurovirologyAug 29, 2026

Using CRISPR to Lower HTLV-1 Virus Levels in Blood Cells from HAM/TSP Patients

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Updated Aug 30, 2026

Abstract

CRISPR treatment achieved a over 50% reduction in proviral loads in PBMCs from HAM/TSP patients five days post-electroporation.

CRISPR technology can excise the HTLV-1 genome in vitro.
Single treatment with CRISPR-RNP complexes targeting specific HTLV-1 sequences resulted in the removal of a 2613 bp segment of the virus's genome.
No detectable off-target activity was observed following CRISPR treatment.
This approach may represent a potential therapeutic strategy for eliminating HTLV-1 DNA from infected cells.

Simplified

CRISPR technology is emerging as a promising therapeutic approach for eliminating chronic viral infections, such as herpesviruses and HIV. Here, for the first time, we demonstrate in vitro that CRISPR can be used to excise the HTLV-1 genome and reduce proviral loads in PBMCs from HAM/TSP (HTLV-1-associated myelopathy/tropical spastic paraparesis) patients. Single treatment with CRISPR-RNP (ribonucleoprotein) complexes composed of two gRNAs targeting the HTLV-1 env gene and 3'LTR sequences resulted in excision of a 2613 bp segment of the proviral genome, spanning tax and HBZ genes, without detectable off-target activity. Furthermore, CRISPR treatment led to over 50% reduction in proviral loads 5 days post-electroporation. Our data indicate that CRISPR-Cas9 gene editing can be used as a therapeutic strategy to eliminate HTLV-1 DNA from infected cells and may serve as a platform for curing HAM/TSP.

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Using CRISPR to Lower HTLV-1 Virus Levels in Blood Cells from HAM/TSP Patients

Abstract

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