ArticleMolecular therapy : the journal of the American Society of Gene Therapy2018
Correction of the Marfan Syndrome Pathogenic FBN1 Mutation by Base Editing in Human Cells and Heterozygous Embryos.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 84 papers.
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Who cites it
84 citing papers in PubMed.
- Article
- Base editing for precision therapeutics.Cell genomics · 2026Review
- Research progress of base editing and prime editing tools based on the CRISPR/Cas system.Molecular therapy. Nucleic acids · 2025Review
- CRISPR-based functional genomics tools in vertebrate models.Experimental & molecular medicine · 2025Review
- The potential of CRISPR-Cas9 in cardiovascular medicine: a focus on hereditary cardiomyopathies.Annals of medicine and surgery (2012) · 2025Article
- Therapeutic Opportunities of Marfan Syndrome: Current Perspectives.Drug design, development and therapy · 2025Review
- Precision Genetic Therapies: Balancing Risk and Benefit in Patients with Heart Failure.Current cardiology reports · 2024Review
- An adenine base editor variant expands context compatibility.Nature biotechnology · 2024Article
- Navigating the CRISPR/Cas Landscape for Enhanced Diagnosis and Treatment of Wilson's Disease.Cells · 2024Review
- The double whammy of ER-retention and dominant-negative effects in numerous autosomal dominant diseases: significance in disease mechanisms and therapy.Journal of biomedical science · 2024Review
- Embryo and fetal gene editing: Technical challenges and progress toward clinical applications.Molecular therapy. Methods & clinical development · 2024Review
- CRISPR/Cas9 as a Mutagenic Factor.International journal of molecular sciences · 2024Review
- Prediction of base editor off-targets by deep learning.Nature communications · 2023Article
- Genomic Innovation in Early Life Cardiovascular Disease Prevention and Treatment.Circulation research · 2023Review
- Human 8-cell embryos enable efficient induction of disease-preventive mutations without off-target effect by cytosine base editor.Protein & cell · 2023Article
- A Review on Advanced CRISPR-Based Genome-Editing Tools: Base Editing and Prime Editing.Molecular biotechnology · 2023Review
- Base editors: development and applications in biomedicine.Frontiers of medicine · 2023Review
- Functional characterization of human genomic variation linked to polygenic diseases.Trends in genetics : TIG · 2023Review
- Review
- Enabling Precision Medicine with CRISPR-Cas Genome Editing Technology: A Translational Perspective.Advances in experimental medicine and biology · 2023Article
24 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
There are urgent demands for efficient treatment of heritable genetic diseases. The base editing technology has displayed its efficiency and precision in base substitution in human embryos, providing a potential early-stage treatment for genetic diseases. Taking advantage of this technology, we corrected a Marfan syndrome pathogenic mutation, FBN1
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Registered trials
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