ArticleMolecular therapy. Nucleic acids2022
Genome editing-mediated knock-in of therapeutic genes ameliorates the disease phenotype in a model of hemophilia.
Article in Molecular therapy. Nucleic acids, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
What it found
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Who cites it
13 citing papers in PubMed, 15 citations in OpenAlex.
- Exploring gene editing as a potential therapeutic strategy for hemophilia.Frontiers in bioengineering and biotechnology · 2026Review
- Advancements in CRISPR-basedFrontiers in genome editing · 2026Review
- Mechanistic and therapeutic dimensions of DcR3-mediated immunomodulation in sepsis.Frontiers in immunology · 2025Review
- CRISPR and gene editing technologies for bleeding disorders.Therapeutic advances in hematology · 2025Review
- Recent Advances in Gene Therapy for Hemophilia: Projecting the Perspectives.Biomolecules · 2024Review
- Targeted Gene Insertion: The Cutting Edge of CRISPR Drug Development with Hemophilia as a Highlight.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2024Review
- In vivo LNP-CRISPR Approaches for the Treatment of Hemophilia.Molecular diagnosis & therapy · 2024Review
- Recent advances in CRISPR-Cas9-based genome insertion technologies.Molecular therapy. Nucleic acids · 2024Review
- Article
- Innovations in RNA therapy for hemophilia.Blood · 2023Review
- Article
- Review
- Expanding CRISPR repertoire using CjCas9 as a smaller editing tool.Molecular therapy. Nucleic acids · 2022Article
Corrections and comments
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Authors and funding
14 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Recently, clinical trials of adeno-associated virus-mediated replacement therapy have suggested long-term therapeutic effects for several genetic diseases of the liver, including hemophilia. However, there remain concerns regarding decreased therapeutic effects when the liver is regenerated or when physiological proliferation occurs. Although genome editing using the clustered regularly interspaced short palindromic repeats/Cas9 system provides an opportunity to solve this problem, low knock-in efficiency may limit its application for therapeutically relevant expression. Here, we identified a novel gene,
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Registered trials
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