ReviewHuman genetics2016
Genome-editing technologies for gene correction of hemophilia.
Review in Human genetics, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 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
24 citing papers in PubMed, 37 citations in OpenAlex.
- Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.Blood · 2018Trial
- Exploring gene editing as a potential therapeutic strategy for hemophilia.Frontiers in bioengineering and biotechnology · 2026Review
- Rapid whole genome sequencing in newborn screening for metabolic diseases.Frontiers in pediatrics · 2025Review
- Comprehensive analysis of off-target and on-target effects resulting from liver-directed CRISPR-Cas9-mediated gene targeting with AAV vectors.Molecular therapy. Methods & clinical development · 2024Article
- CRISPR-Cas9 system: a novel and promising era of genotherapy for beta-hemoglobinopathies, hematological malignancy, and hemophilia.Annals of hematology · 2024Review
- CRISPR/Cas9 and Nanotechnology Pertinence in Agricultural Crop Refinement.Frontiers in plant science · 2022Review
- Insights of CRISPR-Cas systems in stem cells: progress in regenerative medicine.Molecular biology reports · 2022Review
- Gene Therapy Approach with an Emphasis on Growth Factors: Theoretical and Clinical Outcomes in Neurodegenerative Diseases.Molecular neurobiology · 2022Review
- CRISPR medicine for blood disorders: Progress and challenges in delivery.Frontiers in genome editing · 2022Review
- Hemophilia a patients with inhibitors: Mechanistic insights and novel therapeutic implications.Frontiers in immunology · 2022Review
- Gene therapy in haemophilia: literature review and regional perspectives for Turkey.Therapeutic advances in hematology · 2022Review
- CRISPR/Cas based gene editing: marking a new era in medical science.Molecular biology reports · 2021Review
- CRISPR-Cas-mediated chromosome engineering for crop improvement and synthetic biology.Nature plants · 2021Review
- Genome Editing in Medicine: Tools and Challenges.Acta medica Lituanic · 2021Review
- Advances in gene therapy for hemophilia.Journal of biosciences · 2020Review
- Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII.Scientific reports · 2019Article
- CCR5 editing by Staphylococcus aureus Cas9 in human primary CD4Retrovirology · 2019Article
- Update on clinical gene therapy for hemophilia.Blood · 2019Review
- In Vivo Genome Editing as a Therapeutic Approach.International journal of molecular sciences · 2018Review
- Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX.Cell reports · 2018Article
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
No grant is acknowledged in the PubMed record.
Abstract
Hemophilia is caused by various mutations in blood coagulation factor genes, including factor VIII (FVIII) and factor IX (FIX), that encode key proteins in the blood clotting pathway. Although the addition of therapeutic genes or infusion of clotting factors may be used to remedy hemophilia's symptoms, no permanent cure for the disease exists. Moreover, patients often develop neutralizing antibodies or experience adverse effects that limit the therapy's benefits. However, targeted gene therapy involving the precise correction of these mutated genes at the genome level using programmable nucleases is a promising strategy. These nucleases can induce double-strand breaks (DSBs) on genomes, and repairs of such induced DSBs by the two cellular repair systems enable a targeted gene correction. Going beyond cultured cell systems, we are now entering the age of direct gene correction in vivo using various delivery tools. Here, we describe the current status of in vivo and ex vivo genome-editing technology related to potential hemophilia gene correction and the prominent issues surrounding its application in patients with monogenic diseases.
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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.