ArticleNature communications2021
Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases.
Article in Nature communications, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
29 citing papers in PubMed, 55 citations in OpenAlex.
- CRISPR-Cas9-based therapies for Huntington's disease and Friedreich's ataxia: mechanisms, advances, and future perspectives.Neurogenetics · 2026Review
- Investigating the molecular mechanisms underlying the anti-CRISPR function of AcrIIA13b protein.The FEBS journal · 2026Article
- And… cut! - how conformational regulation of CRISPR-Cas effectors directs nuclease activity.The Biochemical journal · 2025Review
- Recent advances in therapeutic gene-editing technologies.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Advancements in CRISPR/Cas systems for disease treatment.Acta pharmaceutica Sinica. B · 2025Review
- Review
- CRISPR: fundamental principles and implications for anaesthesia.British journal of anaesthesia · 2025Review
- CRISPR/Cas9: a sustainable technology to enhance climate resilience in major Staple Crops.Frontiers in genome editing · 2025Review
- PreBP: an interpretable, optimized ensemble framework using routine complete blood count for rapid pathogen identification in bacterial pneumonia.Frontiers in bioinformatics · 2025Article
- Engineered PsCas9 enables therapeutic genome editing in mouse liver with lipid nanoparticles.Nature communications · 2024Article
- PAM-flexible Engineered FnCas9 variants for robust and ultra-precise genome editing and diagnostics.Nature communications · 2024Article
- Engineering of a compact, high-fidelity EbCas12a variant that can be packaged with its crRNA into an all-in-one AAV vector delivery system.PLoS biology · 2024Article
- Transposons and CRISPR: Rewiring Gene Editing.Biochemistry · 2023Article
- Advances in Recombinant Adeno-Associated Virus Vectors for Neurodegenerative Diseases.Biomedicines · 2023Review
- Inhibitory mechanism of CRISPR-Cas9 by AcrIIC4.Nucleic acids research · 2023Article
- CRISPR-Cas System, a Possible "Savior" of Rice Threatened by Climate Change: An Updated Review.Rice (New York, N.Y.) · 2023Review
- Genome editing and cancer therapy: handling the hypoxia-responsive pathway as a promising strategy.Cellular and molecular life sciences : CMLS · 2023Review
- Massively parallel evaluation and computational prediction of the activities and specificities of 17 small Cas9s.Nature methods · 2023Article
- Novel and Engineered Type II CRISPR Systems from Uncultivated Microbes with Broad Genome Editing Capability.The CRISPR journal · 2023Article
- Population-wide gene disruption in the murine lung epithelium via AAV-mediated delivery of CRISPR-Cas9 components.Molecular therapy. Methods & clinical development · 2022Article
Corrections and comments
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Authors and funding
28 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Streptococcus pyogenes (Spy) Cas9 has potential as a component of gene therapeutics for incurable diseases. One of its limitations is its large size, which impedes its formulation and delivery in therapeutic applications. Smaller Cas9s are an alternative, but lack robust activity or specificity and frequently recognize longer PAMs. Here, we investigated four uncharacterized, smaller Cas9s and found three employing a "GG" dinucleotide PAM similar to SpyCas9. Protein engineering generated synthetic RNA-guided nucleases (sRGNs) with editing efficiencies and specificities exceeding even SpyCas9 in vitro and in human cell lines on disease-relevant targets. sRGN mRNA lipid nanoparticles displayed manufacturing advantages and high in vivo editing efficiency in the mouse liver. Finally, sRGNs, but not SpyCas9, could be packaged into all-in-one AAV particles with a gRNA and effected robust in vivo editing of non-human primate (NHP) retina photoreceptors. Human gene therapy efforts are expected to benefit from these improved alternatives to existing CRISPR nucleases.
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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.