ReviewPhysiological reviews2018
Myoediting: Toward Prevention of Muscular Dystrophy by Therapeutic Genome Editing.
Review in Physiological reviews, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
28 citing papers in PubMed, 1 synthesis or guideline pooled it, 40 citations in OpenAlex.
- Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future.Pharmacological reports : PR · 2020Pooled it
- Single-cut gene therapy in a one-step generated rhesus monkey model of Duchenne muscular dystrophy.Cell reports. Medicine · 2025Article
- Advancing CRISPR genome editing into gene therapy clinical trials: progress and future prospects.Expert reviews in molecular medicine · 2025Review
- Gene Editing for Duchenne Muscular Dystrophy: From Experimental Models to Emerging Therapies.Degenerative neurological and neuromuscular disease · 2025Review
- Preventive Human Genome Editing and Enhancement: Candidate Criteria for Governance.The Hastings Center report · 2024Article
- In vivo adenine base editing rescues adrenoleukodystrophy in a humanized mouse model.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- [How safe is gene therapy? : Second death after Duchenne therapy].Innere Medizin (Heidelberg, Germany) · 2024Review
- Efficient Gene Editing for Heart Disease via ELIP-Based CRISPR Delivery System.Pharmaceutics · 2024Article
- Targeting Duchenne muscular dystrophy by skipping DMD exon 45 with base editors.Molecular therapy. Nucleic acids · 2023Article
- CRISPR-Editing Therapy for Duchenne Muscular Dystrophy.Human gene therapy · 2023Review
- Genome Editing to Abrogate Muscle Atrophy.Advances in experimental medicine and biology · 2023Review
- CRISPR-Cas9 Correction of Duchenne Muscular Dystrophy in Mice by a Self-Complementary AAV Delivery System.Methods in molecular biology (Clifton, N.J.) · 2023Article
- Review
- A humanized knockin mouse model of Duchenne muscular dystrophy and its correction by CRISPR-Cas9 therapeutic gene editing.Molecular therapy. Nucleic acids · 2022Article
- Utilization and Potential of RNA-Based Therapies in Cardiovascular Disease.JACC. Basic to translational science · 2022Review
- CRISPR Modeling and Correction of Cardiovascular Disease.Circulation research · 2022Review
- CRISPR Therapeutics for Duchenne Muscular Dystrophy.International journal of molecular sciences · 2022Review
- Toward CRISPR Therapies for Cardiomyopathies.Circulation · 2021Article
- A consolidated AAV system for single-cut CRISPR correction of a common Duchenne muscular dystrophy mutation.Molecular therapy. Methods & clinical development · 2021Article
- Toward the correction of muscular dystrophy by gene editing.Proceedings of the National Academy of Sciences of the United States of America · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 1 institution in 1 country.
Funding
Abstract
Muscular dystrophies represent a large group of genetic disorders that significantly impair quality of life and often progress to premature death. There is no effective treatment for these debilitating diseases. Most therapies, developed to date, focus on alleviating the symptoms or targeting the secondary effects, while the underlying gene mutation is still present in the human genome. The discovery and application of programmable nucleases for site-specific DNA double-stranded breaks provides a powerful tool for precise genome engineering. In particular, the CRISPR/Cas system has revolutionized the genome editing field and is providing a new path for disease treatment by targeting the disease-causing genetic mutations. In this review, we provide a historical overview of genome-editing technologies, summarize the most recent advances, and discuss potential strategies and challenges for permanently correcting genetic mutations that cause muscular dystrophies.
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What OpenQuestion holds
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.