Key result
CRISPR-Cas9 targeting HTLV-1 reduces proviral loads by ~50% in PBMCs from HAM/TSP patients.
Why the study?
CRISPR technology is emerging as a promising therapeutic approach for chronic viral infections, prompting investigation into its ability to excise the HTLV-1 genome and reduce proviral loads in HAM/TSP patients.
Does CRISPR-Cas9 gene editing reduce proviral loads in PBMCs from HAM/TSP patients?
Does CRISPR-Cas9 gene editing reduce proviral loads in PBMCs from HAM/TSP patients?
CRISPR-Cas9 gene editing can successfully excise HTLV-1 DNA and reduce proviral loads in patient-derived PBMCs, offering a potential therapeutic strategy for HAM/TSP.
Supports CRISPR-Cas9 HTLV-1 excision in HAM/TSP PBMCs; hypothesis-generating and leaves open in vivo translation.
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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Brancazio et al. (2026) studied HTLV-1-associated myelopathy/tropical spastic paraparesis (HAM/TSP) (n=10). CRISPR-Cas9 targeting HTLV-1 env and 3'LTR vs. Non-targeting CRISPR-Cas9 was evaluated on Proviral load (PVL) (>50% reduction, p=<0.05). Single treatment with CRISPR-Cas9 targeting the HTLV-1 env gene and 3'LTR sequences resulted in over 50% reduction in proviral loads in PBMCs from HAM/TSP patients 5 days post-treatment.
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