Research graph
References from CRISPR-based genome editing of smooth muscle cells: Disease models, therapeutic applications, and future perspectives. Local targets link to admitted publications; unresolved targets remain external evidence.
A single-cell transcriptomic inventory of murine smooth muscle cells
10.1016/j.devcel.2022.09.015 · 2022 · External reference
Neuromuscular disease of the gastrointestinal tract
10.1097/00000441-199103000-00010 · 1991 · External reference
The epidemiology and pathophysiology of neurogenic bladder
2013 · External reference
Airway smooth muscle in the pathophysiology and treatment of asthma
10.1152/japplphysiol.00950.2012 · 2013 · External reference
Regulation of vascular smooth muscle cell differentiation
10.1016/j.jvs.2007.03.001 · 2007 · External reference
Epigenetic regulation of smooth muscle cell plasticity
10.1016/j.bbagrm.2014.06.004 · 2015 · External reference
MEF2B-Nox1 signaling is critical for stretch-induced phenotypic modulation of vascular smooth muscle cells
10.1161/atvbaha.114.304936 · 2015 · External reference
Role of vascular smooth muscle cell phenotypic switching and calcification in aortic aneurysm formation
10.1161/atvbaha.119.312787 · 2019 · External reference
Smooth muscle cell phenotypic switching in atherosclerosis
10.1093/cvr/cvs115 · 2012 · External reference
CRISPR-Cas9 editing of the HBG1 and HBG2 promoters to treat sickle cell disease
10.1056/nejmoa2215643 · 2023 · External reference
A consolidated AAV system for single-cut CRISPR correction of a common Duchenne muscular dystrophy mutation
10.1016/j.omtm.2021.05.014 · 2021 · External reference
Cardiac Myoediting attenuates cardiac abnormalities in human and mouse models of Duchenne muscular dystrophy
10.1161/circresaha.121.319579 · 2021 · External reference
Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors
10.1038/s41587-020-0561-9 · 2020 · External reference
CRISPR modeling and correction of cardiovascular disease
10.1161/circresaha.122.320496 · 2022 · External reference
Programmable base editing of a•T to G•C in genomic DNA without DNA cleavage
10.1038/nature24644 · 2017 · External reference
Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
10.1038/nature17946 · 2016 · External reference
Efficient C*G-to-G*C base editors developed using CRISPRi screens, target-library analysis, and machine learning
10.1038/s41587-021-00938-z · 2021 · External reference
CRISPR C-to-G base editors for inducing targeted DNA transversions in human cells
10.1038/s41587-020-0609-x · 2021 · External reference
Glycosylase base editors enable C-to-a and C-to-G base changes
10.1038/s41587-020-0592-2 · 2021 · External reference
Adenine transversion editors enable precise, efficient a*T-to-C*G base editing in mammalian cells and embryos
10.1038/s41587-023-01821-9 · 2024 · External reference
Programmable A-to-Y base editing by fusing an adenine base editor with an N-methylpurine DNA glycosylase
10.1038/s41587-022-01595-6 · 2023 · External reference
Development of deaminase-free T-to-S base editor and C-to-G base editor by engineered human uracil DNA glycosylase
10.1038/s41467-024-49343-5 · 2024 · External reference
In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice
10.1038/s41586-020-03086-7 · 2021 · External reference
Precise genomic editing of pathogenic mutations in RBM20 rescues dilated cardiomyopathy
10.1126/scitranslmed.ade1633 · 2022 · External reference
Base editing correction of hypertrophic cardiomyopathy in human cardiomyocytes and humanized mice
10.1038/s41591-022-02176-5 · 2023 · External reference
Ablation of CaMKIIδ oxidation by CRISPR-Cas9 base editing as a therapy for cardiac disease
10.1126/science.ade1105 · 2023 · External reference
Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice
10.1038/s41591-022-02190-7 · 2023 · External reference
Precise gene editing of pathogenic Lamin a mutations corrects cardiac disease
10.1073/pnas.2515267122 · 2025 · External reference
Designing and executing prime editing experiments in mammalian cells
10.1038/s41596-022-00724-4 · 2022 · External reference
Search-and-replace genome editing without double-strand breaks or donor DNA
10.1038/s41586-019-1711-4 · 2019 · External reference
Prime editing: the next frontier in precision gene therapy
10.1002/jgm.70040 · 2025 · External reference
Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice
10.1038/s41551-023-01026-0 · 2023 · External reference
Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cells
2024 · External reference
A mediator complex subunit 12 gain-of-function mutation induces partial leiomyoma cell properties in human uterine smooth muscle cells
2022 · External reference
Genome editing in cardiovascular biology
10.1161/circresaha.116.310197 · 2017 · External reference
Harnessing iPSCs, 3D organoids, and multiomics to model rare vascular diseases: emerging new approach methodologies
10.1177/1358863x251394285 · 2026 · External reference
An iPSC-derived vascular model of Marfan syndrome identifies key mediators of smooth muscle cell death
10.1038/ng.3723 · 2017 · External reference
hiPSC modeling of lineage-specific smooth muscle cell defects caused by TGFBR1(A230T) variant, and its therapeutic implications for Loeys-Dietz syndrome
10.1161/circulationaha.121.054744 · 2021 · External reference
Generation of an induced pluripotent stem cell and isogenic control line from a vascular Ehlers-Danlos syndrome (vEDS) patient harboring a pathogenic c.755G>T in the COL3A1 gene
2026 · External reference
Loss of LMOD1 impairs smooth muscle cytocontractility and causes megacystis microcolon intestinal hypoperistalsis syndrome in humans and mice
10.1073/pnas.1620507114 · 2017 · External reference
Familial megacystis microcolon intestinal hypoperistalsis syndrome: a systematic review
10.1007/s00383-013-3357-x · 2013 · External reference
Loss of the coronary artery disease risk gene LMOD1 in vascular smooth muscle cells triggers rapid-onset coronary atherosclerosis
10.1161/circulationaha.126.080105 · 2026 · External reference
Heterozygous Actg2(R257C) mice mimic the phenotype of megacystis microcolon intestinal hypoperistalsis syndrome
10.1111/nmo.14472 · 2023 · External reference
Determining the pathogenicity of a genomic variant of uncertain significance using CRISPR/Cas9 and human-induced pluripotent stem cells
10.1161/circulationaha.117.032273 · 2018 · External reference
Correction of a CADASIL point mutation using adenine base editors in hiPSCs and blood vessel organoids
10.1016/j.jgg.2023.04.013 · 2024 · External reference
Adenine base editing rescues pathogenic phenotypes in tissue engineered vascular model of Hutchinson-Gilford progeria syndrome
10.1063/5.0244026 · 2025 · External reference
Bioengineered vascular grafts with a pathogenic TGFBR1 variant model aneurysm formation in vivo and reveal underlying collagen defects
10.1126/scitranslmed.adg6298 · 2024 · External reference
CRISPR/Cas9 delivery mediated with hydroxyl-rich Nanosystems for gene editing in aorta
10.1002/advs.201900386 · 2019 · External reference
Mutations in smooth muscle alpha-actin (ACTA2) cause coronary artery disease, stroke, and Moyamoya disease, along with thoracic aortic disease
10.1016/j.ajhg.2009.04.007 · 2009 · External reference
De novo ACTA2 mutation causes a novel syndrome of multisystemic smooth muscle dysfunction
10.1002/ajmg.a.33657 · 2010 · External reference
The defining pathology of the new clinical and histopathologic entity ACTA2-related cerebrovascular disease
10.1186/s40478-015-0262-7 · 2015 · External reference
Aortic disease presentation and outcome associated with ACTA2 mutations
10.1161/circgenetics.114.000943 · 2015 · External reference
Expanding ACTA2 genotypes with corresponding phenotypes overlapping with smooth muscle dysfunction syndrome
10.1002/ajmg.a.62775 · 2022 · External reference
Genomic editing of a pathogenic sequence variant in ACTA2 rescues multisystemic smooth muscle dysfunction syndrome in mice
10.1161/circulationaha.125.074218 · 2025 · External reference
Treatment of a severe vascular disease using a bespoke CRISPR-Cas9 base editor in mice
10.1038/s41551-025-01499-1 · 2026 · External reference
Engineered materials for in vivo delivery of genome-editing machinery
10.1038/s41578-019-0145-9 · 2019 · External reference
The promise and challenge of therapeutic genome editing
10.1038/s41586-020-1978-5 · 2020 · External reference
Lethal immunotoxicity in high-dose systemic AAV therapy
10.1016/j.ymthe.2023.10.015 · 2023 · External reference
Deaths in gene therapy of Duchenne muscular dystrophy and other diseases: underlying mechanisms and mitigating strategies
10.1016/j.ymthe.2025.12.067 · 2026 · External reference
Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies
10.1016/j.omtm.2024.101273 · 2024 · External reference
Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies
10.1172/jci173510 · 2024 · External reference
CD8(+) T-cell responses to adeno-associated virus capsid in humans
10.1038/nm1549 · 2007 · External reference
The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice
10.1172/jci37607 · 2009 · External reference
The effect of CpG sequences on capsid-specific CD8(+) T cell responses to AAV vector gene transfer
10.1016/j.ymthe.2019.11.014 · 2020 · External reference
Lipid nanoparticle Systems for Enabling Gene Therapies
10.1016/j.ymthe.2017.03.013 · 2017 · External reference
Virus-like particle mediated CRISPR/Cas9 delivery for efficient and safe genome editing
10.3390/life10120366 · 2020 · External reference
CRISPR RNA-guided activation of endogenous human genes
10.1038/nmeth.2598 · 2013 · External reference
High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects
10.1038/nature16526 · 2016 · External reference
Rationally engineered Cas9 nucleases with improved specificity
10.1126/science.aad5227 · 2016 · External reference
A highly specific SpCas9 variant is identified by in vivo screening in yeast
10.1038/nbt.4066 · 2018 · External reference
Latest developed strategies to minimize the off-target effects in CRISPR-Cas-mediated genome editing
10.3390/cells9071608 · 2020 · External reference
Improving CRISPR genome editing by engineering guide RNAs
10.1016/j.tibtech.2019.01.009 · 2019 · External reference
Promoters to study vascular smooth muscle
10.1161/atvbaha.119.312449 · 2019 · External reference
Smooth muscle cells differentiated from mesenchymal stem cells are regulated by microRNAs and suitable for vascular tissue grafts
10.1074/jbc.ra118.001739 · 2018 · External reference
Generation and comparative analysis of an Itga8-CreER (T2) mouse with preferential activity in vascular smooth muscle cells
10.1038/s44161-022-00162-1 · 2022 · External reference
An engineered adeno-associated virus capsid mediates efficient transduction of Pericytes and smooth muscle cells of the brain vasculature
10.1089/hum.2022.211 · 2023 · External reference
Programmable lipid nanoparticles for RNA therapeutics: design principles and clinical translation
2026 · External reference
Patient-specific in vivo gene editing to treat a rare genetic disease
10.1056/nejmoa2504747 · 2025 · External reference
Elimination of CaMKIIδ autophosphorylation by CRISPR-Cas9 base editing improves survival and cardiac function in heart failure in mice
10.1161/circulationaha.123.065117 · 2023 · External reference
CRISPR-Cas9 base editing of pathogenic CaMKIIδ improves cardiac function in a humanized mouse model
10.1172/jci175164 · 2024 · External reference
Ablation of PKCalpha phosphorylation by CRISPR-Cas9 base editing rescues heart failure
10.1161/circresaha.125.326738 · 2026 · External reference
RNA-guided gene activation by CRISPR-Cas9-based transcription factors
10.1038/nmeth.2600 · 2013 · External reference
Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers
10.1038/nbt.3199 · 2015 · External reference
CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes
10.1016/j.cell.2013.06.044 · 2013 · External reference
