ArticleCell stem cell2011
Targeted gene correction of laminopathy-associated LMNA mutations in patient-specific iPSCs.
Article in Cell stem cell, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 144 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
144 citing papers in PubMed, 217 citations in OpenAlex.
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- Prevalent integration of genomic repetitive and regulatory elements and donor sequences at CRISPR-Cas9-induced breaks.Communications biology · 2025Article
- Review
- Navigating Lipodystrophy: Insights from Laminopathies and Beyond.International journal of molecular sciences · 2024Review
- Stem cell modeling of nervous system tumors.Disease models & mechanisms · 2024Review
- Trichostatin A for Efficient CRISPR-Cas9 Gene Editing of Human Pluripotent Stem Cells.The CRISPR journal · 2023Article
- The Molecular and Cellular Basis of Hutchinson-Gilford Progeria Syndrome and Potential Treatments.Genes · 2023Review
- Achieving single nucleotide sensitivity in direct hybridization genome imaging.Nature communications · 2022Article
- Genetic enhancement: an avenue to combat aging-related diseases.Life medicine · 2022Review
- Rare and undiagnosed diseases: From disease-causing gene identification to mechanism elucidation.Fundamental research · 2022Review
- Wiskott-Aldrich syndrome protein forms nuclear condensates and regulates alternative splicing.Nature communications · 2022Article
- Modeling Nonischemic Genetic Cardiomyopathies Using Induced Pluripotent Stem Cells.Current cardiology reports · 2022Review
- Preclinical Advances of Therapies for Laminopathies.Journal of clinical medicine · 2021Review
- Approaches to characterize the transcriptional trajectory of human myogenesis.Cellular and molecular life sciences : CMLS · 2021Review
- Current Status and Challenges of Stem Cell Treatment for Alzheimer's Disease.Journal of Alzheimer's disease : JAD · 2021Review
- METTL3 counteracts premature aging via m6A-dependent stabilization of MIS12 mRNA.Nucleic acids research · 2020Article
- Generation of a Hutchinson-Gilford progeria syndrome monkey model by base editing.Protein & cell · 2020Article
- Progress and trends in the development of therapies for Hutchinson-Gilford progeria syndrome.Aging cell · 2020Review
- An Omics View of Emery-Dreifuss Muscular Dystrophy.Journal of personalized medicine · 2020Review
- A Single "All-in-One" Helper-Dependent Adenovirus to Deliver Donor DNA and CRISPR/Cas9 for Efficient Homology-Directed Repair.Molecular therapy. Methods & clinical development · 2020Article
84 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
18 authors at 2 institutions in 2 countries.
Funding
Abstract
Combination of stem cell-based approaches with gene-editing technologies represents an attractive strategy for studying human disease and developing therapies. However, gene-editing methodologies described to date for human cells suffer from technical limitations including limited target gene size, low targeting efficiency at transcriptionally inactive loci, and off-target genetic effects that could hamper broad clinical application. To address these limitations, and as a proof of principle, we focused on homologous recombination-based gene correction of multiple mutations on lamin A (LMNA), which are associated with various degenerative diseases. We show that helper-dependent adenoviral vectors (HDAdVs) provide a highly efficient and safe method for correcting mutations in large genomic regions in human induced pluripotent stem cells and can also be effective in adult human mesenchymal stem cells. This type of approach could be used to generate genotype-matched cell lines for disease modeling and drug discovery and potentially also in therapeutics.
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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.