ArticleScience (New York, N.Y.)2024
In vivo editing of lung stem cells for durable gene correction in mice.
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Article in Science (New York, N.Y.), 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 100 papers.
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100citing papers in PubMed
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Who cites it
100 citing papers in PubMed.
- Delivering the blueprint: Advances and challenges in mRNA therapeutics for the respiratory system.International journal of pharmaceutics: X · 2026Review
- Lipid nanoparticles optimized for large RNA cargo and tissue targeting enhance in vivo genome editing.Nature biotechnology · 2026Article
- Ligand-Mediated Reprogramming Redirects Liver-Tropic Ionizable Lipid Nanoparticles for Lung-Selective mRNA Delivery.Angewandte Chemie (International ed. in English) · 2026Article
- Dense RNA motif modifications enable robust in vivo prime editing and enhance efficiencies of diverse editing systems.Nature biomedical engineering · 2026Article
- Double-stranded DNA donors and CRISPR-Cas9 for universal correction of mutations causing cystic fibrosis in human airway cells.Molecular therapy. Nucleic acids · 2026Article
- Precision chemical engineering of dendrimers for nucleic acid delivery.Nature reviews. Chemistry · 2026Review
- Targeted Therapy for Restoring CFTR Activity: From Experimental to Clinical Features.International journal of molecular sciences · 2026Review
- Article
- Splenic dendritic cell-targeting mRNA transfection of H-type ionizable lipid-based LNPs for enhancing tumor immunotherapy.Bioactive materials · 2026Article
- SORT LNPs encapsulating Cas9 mRNA achieve efficient editing in skeletal muscle in a dystrophic mouse model.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Lipoic Acid Derivative/PEI Composite Nanoparticles Enable Efficient mRNA Delivery via Thiol-Mediated Cellular Uptake.Macromolecular bioscience · 2026Article
- Nanotechnology-mediated precision delivery of mRNA.Nature materials · 2026Review
- Remodeling the pathological airway: advanced nanotechnology for diagnostics and therapeutics in cystic fibrosis.Journal of nanobiotechnology · 2026Review
- Therapeutic adenine base editor with minimized off-target effects.Protein & cell · 2026Article
- The RNA delivery dilemma-lipid versus polymer nanoparticle platforms.Drug delivery and translational research · 2026Article
- Considerations for early life genetic therapies in cystic fibrosis.American journal of physiology. Lung cellular and molecular physiology · 2026Review
- Delivering base editors to the lungs.Nature materials · 2026Article
- Optimized lentivirus-derived virus-like particles for efficient delivery of Cas9-based genome editors.Nucleic acids research · 2026Article
- Selective Elimination of TP53 Mutant Cells by Transcript-Activated Chromatin Shredding.bioRxiv : the preprint server for biology · 2026Article
- Base editing and nanoparticle transfection of airway cell types essential for treatment of cystic fibrosis.JCI insight · 2026Article
40 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
30 authors.
Yehui SunDepartment of Biomedical Engineering, Department of Biochemistry, Simmons Comprehensive Cancer Center, Program in Genetic Drug Engineering, The University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.ORCID 0000-0003-0215-6095
Sumanta ChatterjeeDepartment of Biomedical Engineering, Department of Biochemistry, Simmons Comprehensive Cancer Center, Program in Genetic Drug Engineering, The University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.ORCID 0000-0002-6832-0073
Xizhen LianDepartment of Biomedical Engineering, Department of Biochemistry, Simmons Comprehensive Cancer Center, Program in Genetic Drug Engineering, The University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
Zachary TraylorDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH 44106, USA.
Sandhya R SattirajuReCode Therapeutics, Menlo Park, CA 94025, USA.ORCID 0009-0005-9824-8294
Yufen XiaoDepartment of Biomedical Engineering, Department of Biochemistry, Simmons Comprehensive Cancer Center, Program in Genetic Drug Engineering, The University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.ORCID 0000-0002-5604-7479
Sean A DilliardDepartment of Biomedical Engineering, Department of Biochemistry, Simmons Comprehensive Cancer Center, Program in Genetic Drug Engineering, The University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
Yun-Chieh SungDepartment of Biomedical Engineering, Department of Biochemistry, Simmons Comprehensive Cancer Center, Program in Genetic Drug Engineering, The University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.ORCID 0000-0001-7525-0694
Minjeong KimDepartment of Biomedical Engineering, Department of Biochemistry, Simmons Comprehensive Cancer Center, Program in Genetic Drug Engineering, The University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
Sang M LeeDepartment of Biomedical Engineering, Department of Biochemistry, Simmons Comprehensive Cancer Center, Program in Genetic Drug Engineering, The University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.ORCID 0000-0001-7658-6229
Stephen MooreDepartment of Biomedical Engineering, Department of Biochemistry, Simmons Comprehensive Cancer Center, Program in Genetic Drug Engineering, The University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.ORCID 0000-0003-4156-5350
Xu WangDepartment of Biomedical Engineering, Department of Biochemistry, Simmons Comprehensive Cancer Center, Program in Genetic Drug Engineering, The University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
Di ZhangDepartment of Biomedical Engineering, Department of Biochemistry, Simmons Comprehensive Cancer Center, Program in Genetic Drug Engineering, The University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.ORCID 0000-0001-5586-1527
Shiying WuDepartment of Biomedical Engineering, Department of Biochemistry, Simmons Comprehensive Cancer Center, Program in Genetic Drug Engineering, The University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.ORCID 0000-0002-6902-9544
Pratima BasakDepartment of Biomedical Engineering, Department of Biochemistry, Simmons Comprehensive Cancer Center, Program in Genetic Drug Engineering, The University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.ORCID 0009-0008-6547-8896
Jialu WangReCode Therapeutics, Menlo Park, CA 94025, USA.ORCID 0009-0000-0758-0034
Jing LiuReCode Therapeutics, Menlo Park, CA 94025, USA.
Rachel J MannDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH 44106, USA.ORCID 0009-0007-4558-2947
David F LePageDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH 44106, USA.ORCID 0009-0008-3820-0645
Weihong JiangDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH 44106, USA.ORCID 0009-0009-1697-1702
Shadaan AbidDepartment of Internal Medicine, The University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
Mirko HennigReCode Therapeutics, Menlo Park, CA 94025, USA.ORCID 0009-0009-0238-737X
Anna MartinezReCode Therapeutics, Menlo Park, CA 94025, USA.ORCID 0000-0001-7245-9687
Brandon A WustmanReCode Therapeutics, Menlo Park, CA 94025, USA.
David J LockhartReCode Therapeutics, Menlo Park, CA 94025, USA.
Raksha JainDepartment of Internal Medicine, The University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
Ronald A ConlonDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH 44106, USA.
Mitchell L DrummDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH 44106, USA.ORCID 0000-0003-4739-5153
Craig A HodgesDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH 44106, USA.ORCID 0000-0003-3332-9024
Daniel J SiegwartDepartment of Biomedical Engineering, Department of Biochemistry, Simmons Comprehensive Cancer Center, Program in Genetic Drug Engineering, The University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.ORCID 0000-0003-3823-1931
Funding
UT Southwestern Medical Center Simmons Comprehensive Cancer CenterP30CA142543 · NCI · UT SOUTHWESTERN MEDICAL CENTER · PI Kathryn Ann O'Donnell · 2010 to 2026
$53.7MDefining the molecular interactions within nanoparticles that enable delivery of long nucleic acidsR01EB025192 · NIBIB · UT SOUTHWESTERN MEDICAL CENTER · PI SIEGWART, DANIEL JOHN · 2018 to 2025
$3.1MNCI NIH HHS P30 CA142543NIBIB NIH HHS R01 EB025192
Abstract
In vivo genome correction holds promise for generating durable disease cures; yet, effective stem cell editing remains challenging. In this work, we demonstrate that optimized lung-targeting lipid nanoparticles (LNPs) enable high levels of genome editing in stem cells, yielding durable responses. Intravenously administered gene-editing LNPs in activatable tdTomato mice achieved >70% lung stem cell editing, sustaining tdTomato expression in >80% of lung epithelial cells for 660 days. Addressing cystic fibrosis (CF), NG-ABE8e messenger RNA (mRNA)-sgR553X LNPs mediated >95% cystic fibrosis transmembrane conductance regulator (CFTR) DNA correction, restored CFTR function in primary patient-derived bronchial epithelial cells equivalent to Trikafta for F508del, corrected intestinal organoids and corrected R553X nonsense mutations in 50% of lung stem cells in CF mice. These findings introduce LNP-enabled tissue stem cell editing for disease-modifying genome correction.
Indexed as
Cystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorGene EditingLiposomesLungNanoparticlesStem CellsAnimalsCRISPR-Cas SystemsEpithelial CellsGenetic TherapyHumansMiceOrganoidsCFTR protein, humanCystic Fibrosis Transmembrane Conductance RegulatorLipid NanoparticlesLiposomes
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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.