ArticleGene therapy2016
Excision of HIV-1 DNA by gene editing: a proof-of-concept in vivo study.
Article in Gene therapy, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 112 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
112 citing papers in PubMed, 202 citations in OpenAlex.
- CRISPR-Cas and Infectious Diseases: A Decade of Translational Advances in Molecular Biotechnology.Biochemical genetics · 2026Review
- HIV cure: from early therapies to cutting-edge strategies.Journal of neurovirology · 2026Review
- Advancing Antiviral Design: Integrating Natural Products, Computation and Targeted Delivery.Chemical biology & drug design · 2026Review
- CRISPR/Cas9-Based Genome Editing: Understanding Differences in DNA Repair Pathways, Profiles, and Outcomes.International journal of molecular sciences · 2026Review
- From mechanism to medicine: CRISPR‒Cas9 delivery strategies, therapeutic applications and translation challenges.Discover nano · 2026Review
- Bipolar CD4-targeted dual-DARPin-55/57 lipid nanoparticle enables efficient CRISPR/Cas-mediated HIV-1 DNA excision and reactivation blockade in latent CD4 T cell lines.Materials today. Bio · 2026Article
- Breaking into HIV-1's Epigenetic Vault: Cure Strategies to Eliminate the Viral Reservoir.Viruses · 2026Review
- CRISPR Applications in HIV Management - Prevention, Diagnosis, Monitoring and Treatment.Current HIV/AIDS reports · 2026Review
- Targeted Drug Delivery Strategies in Overcoming Antimicrobial Resistance: Advances and Future Directions.Pharmaceutics · 2025Review
- Neuropathogenesis of acute HIV: mechanisms, biomarkers, and therapeutic approaches.Current opinion in HIV and AIDS · 2025Review
- From Origin to the Present: Establishment, Mechanism, Evolutions and Biomedical Applications of the CRISPR/Cas-Based Macromolecular System in Brief.Molecules (Basel, Switzerland) · 2025Review
- AAV-mediated gene therapies by miniature gene editing tools.Science China. Life sciences · 2024Review
- Viral and nonviral nanocarriers forNano research · 2024Article
- High-resolution Inference of Multiplexed Anti-HIV Gene Editing using Single-Cell Targeted DNA Sequencing.bioRxiv : the preprint server for biology · 2024Article
- Precision in Action: The Role of Clustered Regularly Interspaced Short Palindromic Repeats/Cas in Gene Therapies.Vaccines · 2024Review
- Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates.Gene therapy · 2024Article
- Genetically encoding multiple functionalities into extracellular vesicles for the targeted delivery of biologics to T cells.Nature biomedical engineering · 2024Article
- HIV-1 mRNA knockdown with CRISPR/CAS9 enhances neurocognitive function.Journal of neurovirology · 2024Article
- Delivering CRISPR to the HIV-1 reservoirs.Frontiers in microbiology · 2024Review
- Low prevalence of HIV in the northern Cameroon: contribution of some AIDS restriction genes and potential implications for gene therapy.Frontiers in genetics · 2024Article
52 more citing papers are in PubMed but not listed here.
Corrections and comments
- Erratum issued
Authors and funding
11 authors at 4 institutions in 2 countries.
Funding
Abstract
A CRISPR/Cas9 gene editing strategy has been remarkable in excising segments of integrated HIV-1 DNA sequences from the genome of latently infected human cell lines and by introducing InDel mutations, suppressing HIV-1 replication in patient-derived CD4+ T-cells, ex vivo. Here, we employed a short version of the Cas9 endonuclease, saCas9, together with a multiplex of guide RNAs (gRNAs) for targeting the viral DNA sequences within the 5'-LTR and the Gag gene for removing critically important segments of the viral DNA in transgenic mice and rats encompassing the HIV-1 genome. Tail-vein injection of transgenic mice with a recombinant Adeno-associated virus 9 (rAAV9) vector expressing saCas9 and the gRNAs, rAAV:saCas9/gRNA, resulted in the cleavage of integrated HIV-1 DNA and excision of a 978 bp DNA fragment spanning between the LTR and Gag gene in the spleen, liver, heart, lung and kidney as well as in the circulating lymphocytes. Retro-orbital inoculation of rAAV9:saCas9/gRNA in transgenic rats eliminated a targeted segment of viral DNA and substantially decreased the level of viral gene expression in circulating blood lymphocytes. The results from the proof-of-concept studies, for the first time, demonstrate the in vivo eradication of HIV-1 DNA by CRISPR/Cas9 on delivery by an rAAV9 vector in a range of cells and tissues that harbor integrated copies of viral DNA.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.