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References from CRISPR-mediated targeting of the LMNA c.745C>T mutation enhances survival and cardiac function in congenital muscular dystrophy. Local targets link to admitted publications; unresolved targets remain external evidence.
De novo LMNA mutations cause a new form of congenital muscular dystrophy
10.1002/ana.21417 · 2008 · External reference
Congenital muscular dystrophy with dropped head linked to the LMNA gene in a Brazilian cohort
10.1016/j.pediatrneurol.2013.11.010 · 2014 · External reference
Phenotype-Genotype Analysis of Chinese Patients with Early-Onset LMNA-Related Muscular Dystrophy
2015 · External reference
Floppy infant syndrome as a first manifestation of LMNA-related congenital muscular dystrophy
10.1016/j.ejpn.2021.04.005 · 2021 · External reference
International retrospective natural history study of LMNA-related congenital muscular dystrophy
10.1093/braincomms/fcab075 · 2021 · External reference
Molecular Pathology of Laminopathies
10.1146/annurev-pathol-042220-034240 · 2022 · External reference
Lamin A/C Assembly Defects in LMNA-Congenital Muscular Dystrophy Is Responsible for the Increased Severity of the Disease Compared with Emery-Dreifuss Muscular Dystrophy
10.3390/cells9040844 · 2020 · External reference
LMNA-related muscular dystrophy: Identification of variants in alternative genes and personalized clinical translation
10.3389/fgene.2023.1135438 · 2023 · External reference
Clinical spectrum and genetic variations of LMNA-related muscular dystrophies in a large cohort of Chinese patients Genotype-phenotype correlations
10.1136/jmedgenet-2019-106671 · 2021 · External reference
Characterization of cardiac involvement in children with LMNA-related muscular dystrophy
10.3389/fcell.2023.1142937 · 2023 · External reference
Clinical features and therapeutic strategies for managing the striated muscle laminopathies
10.1080/21678707.2016.1180975 · 2016 · External reference
Genetic reduction of mTOR extends lifespan in a mouse model of Hutchinson-Gilford Progeria syndrome
10.1111/acel.13457 · 2021 · External reference
Targeting of NAT10 enhances healthspan in a mouse model of human accelerated aging syndrome
10.1038/s41467-018-03770-3 · 2018 · External reference
Inhibition of extracellular signal-regulated kinase 1/2 signaling has beneficial effects on skeletal muscle in a mouse model of Emery-Dreifuss muscular dystrophy caused by lamin A/C gene mutation
10.1186/2044-5040-3-17 · 2013 · External reference
Single-dose CRISPR–Cas9 therapy extends lifespan of mice with Hutchinson–Gilford progeria syndrome
10.1038/s41591-019-0343-4 · 2019 · External reference
Development of a CRISPR/Cas9-based therapy for Hutchinson–Gilford progeria syndrome
10.1038/s41591-018-0338-6 · 2019 · External reference
In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice
10.1038/s41586-020-03086-7 · 2021 · External reference
Mechanisms of angiogenic incompetence in Hutchinson-Gilford progeria via downregulation of endothelial NOS
10.1111/acel.13388 · 2021 · External reference
Transient expression of an adenine base editor corrects the Hutchinson-Gilford progeria syndrome mutation and improves the skin phenotype in mice
10.1038/s41467-022-30800-y · 2022 · External reference
LMNA Co-Regulated Gene Expression as a Suitable Readout after Precise Gene Correction
2022 · 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
Adenine base editor-based correction of the cardiac pathogenic Lmna c.1621C > T mutation in murine hearts
2024 · External reference
Precise gene editing of pathogenic Lamin A mutations corrects cardiac disease
10.1073/pnas.2515267122 · 2025 · External reference
Gene Therapy via Trans-Splicing for LMNA-Related Congenital Muscular Dystrophy
10.1016/j.omtn.2017.12.012 · 2018 · External reference
DNA targeting specificity of RNA-guided Cas9 nucleases
10.1038/nbt.2647 · 2013 · External reference
Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9
10.1038/nbt.3437 · 2016 · External reference
Structure and specificity of the RNA-guided endonuclease Cas9 during DNA interrogation, target binding and cleavage
10.1093/nar/gkv892 · 2015 · External reference
Modeling Skeletal Muscle Laminopathies Using Human Induced Pluripotent Stem Cells Carrying Pathogenic LMNA Mutations
10.3389/fphys.2018.01332 · 2018 · External reference
Loss of A-type lamin expression compromises nuclear envelope integrity leading to muscular dystrophy
10.1083/jcb.147.5.913 · 1999 · External reference
CRISPRs for optimal targeting: Delivery of CRISPR components as DNA, RNA, and protein into cultured cells and single-cell embryos
10.1089/hum.2016.009 · 2016 · External reference
How specific is CRISPR/Cas9 really?
10.1016/j.cbpa.2015.10.001 · 2015 · External reference
Multiplex Genome Engineering Using CRISPR/Cas Systems
10.1126/science.1231143 · 2013 · External reference
High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells
10.1038/nbt.2623 · 2013 · External reference
Interference by clustered regularly interspaced short palindromic repeat (CRISPR) RNA is governed by a seed sequence
10.1073/pnas.1104144108 · 2011 · External reference
Real-time observation of DNA recognition and rejection by the RNA-guided endonuclease Cas9
10.1038/ncomms12778 · 2016 · External reference
Genome-wide binding of the CRISPR endonuclease Cas9 in mammalian cells
10.1038/nbt.2889 · 2014 · External reference
Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity
10.1016/j.cell.2013.08.021 · 2013 · External reference
Enhanced specificity and efficiency of the CRISPR/Cas9 system with optimized sgRNA parameters in Drosophila
10.1016/j.celrep.2014.09.044 · 2014 · External reference
Unraveling LMNA Mutations in Metabolic Syndrome: Cellular Phenotype and Clinical Pitfalls
10.3390/cells9020310 · 2020 · External reference
Consequences of lmna exon 4 mutations in myoblast function
10.3390/cells9051286 · 2020 · External reference
Nuclear envelope defects associated with LMNA mutations cause dilated cardiomyopathy and Emery-Dreifuss muscular dystrophy
10.1242/jcs.114.24.4447 · 2001 · External reference
Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice
10.1038/s41591-022-02190-7 · 2023 · External reference
Dynamics of Cell Generation and Turnover in the Human Heart
10.1016/j.cell.2015.05.026 · 2015 · External reference
Study of non-muscle cells of the adult mammalian heart: a fine structural analysis and distribution
1980 · External reference
Improving CrIsPr-Cas nuclease specificity using truncated guide rNAs
10.1038/nbt.2808 · 2014 · External reference
Optimizing sgRNA length to improve target specificity and efficiency for the GGTA1 gene using the CRISPR/Cas9 gene editing system
10.1371/journal.pone.0226107 · 2019 · External reference
Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy
10.1126/scitranslmed.aan8081 · 2017 · External reference
Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
10.1126/science.aau1549 · 2018 · External reference
Long-term maintenance of dystrophin expression and resistance to injury of skeletal muscle in gene edited DMD mice
10.1016/j.omtn.2022.03.004 · 2022 · External reference
CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells
10.1126/sciadv.aav4324 · 2019 · External reference
A humanized knockin mouse model of Duchenne muscular dystrophy and its correction by CRISPR-Cas9 therapeutic gene editing
10.1016/j.omtn.2022.07.024 · 2022 · External reference
Modulation of LMNA splicing as a strategy to treat prelamin A diseases
10.1172/jci85908 · 2016 · External reference
Splicing-directed therapy in a new mouse model of human accelerated aging
10.1126/scitranslmed.3002847 · 2011 · External reference
Ameliorating pathogenesis by removing an exon containing a missense mutation: a potential exon-skipping therapy for laminopathies
10.1038/gt.2015.8 · 2015 · External reference
Mutation analysis of the lamin A/C gene (LMNA) among patients with different cardiomuscular phenotypes
10.1136/jmg.40.12.e132 · 2003 · External reference
Chapter 7 Generation and Culture of Mouse Embryonic Fibroblasts
10.1007/978-1-4939-9167-9_7 · 2019 · External reference
Breaking-Cas—interactive design of guide RNAs for CRISPR-Cas experiments for ENSEMBL genomes
10.1093/nar/gkw407 · 2016 · External reference
Genome engineering using the CRISPR-Cas9 system
10.1038/nprot.2013.143 · 2013 · External reference
Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice
10.1016/j.ymthe.2020.06.021 · 2020 · External reference
Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts
2016 · External reference
