ArticleJournal of the American Chemical Society2018
Receptor-Mediated Delivery of CRISPR-Cas9 Endonuclease for Cell-Type-Specific Gene Editing.
Article in Journal of the American Chemical Society, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 69 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
69 citing papers in PubMed, 153 citations in OpenAlex.
- Non-Viral CRISPR carriers: transient delivery with lasting effects.Drug delivery · 2026Review
- CRISPR-Cas9 in Cancer Therapy: Overcoming Delivery Barriers and Off-Target Effects for Clinical Translation.Health science reports · 2026Article
- Protein Modifications for Cellular Protein Delivery.Chemical reviews · 2026Review
- Nanoengineering Systems for Gene Therapy: Mechanisms, Modalities, and Future Directions.International journal of molecular sciences · 2026Review
- Calcium Shock Enables Efficient and Programmable Particle Delivery for Genome Editing Applications.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Nature Inspired Delivery Vehicles for CRISPR-Based Genome Editing.Small (Weinheim an der Bergstrasse, Germany) · 2026Review
- An AIEgen-Based Carrier Enables Efficient Cytosolic Delivery of Bioactive Proteins.International journal of nanomedicine · 2026Article
- Peptide-enabled ribonucleoprotein delivery for CRISPR engineering (PERC) in primary human immune cells and hematopoietic stem cells.Nature protocols · 2025Review
- Efficient Direct Cytosolic Protein Delivery via Protein-Linker Co-engineering.ACS applied materials & interfaces · 2025Article
- Precisely Targeted Nanoparticles for CRISPR-Cas9 Delivery in Clinical Applications.Nanomaterials (Basel, Switzerland) · 2025Review
- Dynamic sampling of a surveillance state enables DNA proofreading by Cas9.Cell chemical biology · 2025Article
- Highly efficient CRISPR-mediated genome editing through microfluidic droplet cell mechanoporation.Nature communications · 2024Article
- Targeted nonviral delivery of genome editors in vivo.Proceedings of the National Academy of Sciences of the United States of America · 2024Review
- Minimization of the E. coli ribosome, aided and optimized by community science.Nucleic acids research · 2024Article
- Application of the CRISPR/Cas9 Gene Editing Method for Modulating Antibody Fucosylation in CHO Cells.Methods in molecular biology (Clifton, N.J.) · 2024Article
- Extracellular vesicle-mediated delivery of CRISPR/Cas9 ribonucleoprotein complex targeting proprotein convertase subtilisin-kexin type 9 (Pcsk9) in primary mouse hepatocytes.Journal of extracellular vesicles · 2024Article
- Fire Burn and Cauldron Bubble: What Is in Your Genome Editing Brew?Biochemistry · 2023Review
- Multivalent Targeting of the Asialoglycoprotein Receptor by Virus-Like Particles.Small (Weinheim an der Bergstrasse, Germany) · 2023Article
- Amphipathic Cell-Penetrating Peptide-Aided Delivery of Cas9 RNP for In Vitro Gene Editing and Correction.Pharmaceutics · 2023Article
- A potential paradigm in CRISPR/Cas systems delivery: at the crossroad of microalgal gene editing and algal-mediated nanoparticles.Journal of nanobiotechnology · 2023Review
9 more citing papers are in PubMed but not listed here.
Corrections and comments
- Commented on byTargeting Cas9.2018
Authors and funding
27 authors at 3 institutions in 1 country.
Funding
Abstract
CRISPR-Cas RNA-guided endonucleases hold great promise for disrupting or correcting genomic sequences through site-specific DNA cleavage and repair. However, the lack of methods for cell- and tissue-selective delivery currently limits both research and clinical uses of these enzymes. We report the design and in vitro evaluation of S. pyogenes Cas9 proteins harboring asialoglycoprotein receptor ligands (ASGPrL). In particular, we demonstrate that the resulting ribonucleoproteins (Cas9-ASGPrL RNP) can be engineered to be preferentially internalized into cells expressing the corresponding receptor on their surface. Uptake of such fluorescently labeled proteins in liver-derived cell lines HEPG2 (ASGPr+) and SKHEP (control; diminished ASGPr) was studied by live cell imaging and demonstrates increased accumulation of Cas9-ASGPrL RNP in HEPG2 cells as a result of effective ASGPr-mediated endocytosis. When uptake occurred in the presence of a peptide with endosomolytic properties, we observed receptor-facilitated and cell-type specific gene editing that did not rely on electroporation or the use of transfection reagents. Overall, these in vitro results validate the receptor-mediated delivery of genome-editing enzymes as an approach for cell-selective gene editing and provide a framework for future potential applications to hepatoselective gene editing in vivo.
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What OpenQuestion holds
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.