ArticleAdvanced materials (Deerfield Beach, Fla.)2021
All-In-One Dendrimer-Based Lipid Nanoparticles Enable Precise HDR-Mediated Gene Editing In Vivo.
Article in Advanced materials (Deerfield Beach, Fla.), 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 46 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.
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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
46 citing papers in PubMed, 96 citations in OpenAlex.
- Precision chemical engineering of dendrimers for nucleic acid delivery.Nature reviews. Chemistry · 2026Review
- Programmable Nucleic Acid Sensing in Human Cells Using Circularizable ssDNA.Nature communications · 2026Article
- The RNA delivery dilemma-lipid versus polymer nanoparticle platforms.Drug delivery and translational research · 2026Article
- Dendrimers Improve Apolipoprotein Nanoparticle mRNA Delivery to Immune Cells.Advanced materials (Deerfield Beach, Fla.) · 2026Article
- Design of cationic ionizable lipids for the delivery of therapeutic nucleic acids.Molecular therapy. Methods & clinical development · 2025Review
- Reinforced plant-derived lipid nanoparticles for oral precise epigenome editing in colonic diseases.Science advances · 2025Article
- Limiting endosomal damage sensing reduces inflammation triggered by lipid nanoparticle endosomal escape.Nature nanotechnology · 2025Article
- Recent Advances in mRNA Delivery Systems for Cancer Therapy.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Review
- Nanotechnology-Based Delivery of CRISPR/Cas9 for Cancer Treatment: A Comprehensive Review.Cells · 2025Review
- Advancements in CRISPR/Cas systems for disease treatment.Acta pharmaceutica Sinica. B · 2025Review
- Engineering tripartite gene editing machinery for highly efficient non-viral targeted genome integration.Nature communications · 2025Article
- CRISPR/Cas9 Ribonucleoprotein Delivery Enhanced by Lipo-Xenopeptide Carriers and Homology-Directed Repair Modulators: Insights from Reporter Cell Lines.International journal of molecular sciences · 2025Article
- Precisely Targeted Nanoparticles for CRISPR-Cas9 Delivery in Clinical Applications.Nanomaterials (Basel, Switzerland) · 2025Review
- Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group.Molecular therapy. Nucleic acids · 2025Review
- Inhalable Dry Powders for Lung mRNA Delivery.Advanced healthcare materials · 2024Article
- Article
- Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells.Nature nanotechnology · 2024Article
- In Silico Screening Accelerates Nanocarrier Design for Efficient mRNA Delivery.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2024Article
- Strategies for enhanced gene delivery to the central nervous system.Nanoscale advances · 2024Review
- An Antibody-CRISPR/Cas Conjugate Platform for Target-Specific Delivery and Gene Editing in Cancer.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2024Article
Corrections and comments
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Authors and funding
7 authors at 1 institution in 1 country.
Funding
Abstract
Clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated (Cas) protein gene editing is poised to transform the treatment of genetic diseases. However, limited progress has been made toward precise editing of DNA via homology-directed repair (HDR) that requires careful orchestration of complex steps. Herein, dendrimer-based lipid nanoparticles (dLNPs) are engineered to co-encapsulate and deliver multiple components for in vivo HDR correction. BFP/GFP switchable HEK293 cells with a single Y66H amino acid mutation are employed to assess HDR-mediated gene editing following simultaneous, one-pot delivery of Cas9 mRNA, single-guide RNA, and donor DNA. Molar ratios of individual LNP components and weight ratios of the three nucleic acids are systematically optimized to increase HDR efficiency. Using flow cytometry, fluorescence imaging, and DNA sequencing to quantify editing, optimized 4A3-SC8 dLNPs edit >91% of all cells with 56% HDR efficiency in vitro and >20% HDR efficiency in xenograft tumors in vivo. Due to the all-in-one simplicity and high efficacy, the developed dLNPs offer a promising route toward the gene correction of disease-causing mutations.
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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.