ArticleExpert opinion on orphan drugs
Obstacles and future of gene therapy for hemophilia.
Article in Expert opinion on orphan drugs. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 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
22 citing papers in PubMed, 33 citations in OpenAlex.
- Long-Term Dystrophin Replacement Therapy in Duchenne Muscular Dystrophy Causes Cardiac Inflammation.JACC. Basic to translational science · 2025Article
- Non-viral and viral delivery systems for hemophilia A therapy: recent development and prospects.Annals of hematology · 2024Review
- The Evolution of Hemophilia Therapeutics: An Illustrated Review.Research and practice in thrombosis and haemostasis · 2024Article
- Understanding AAV vector immunogenicity: from particle to patient.Theranostics · 2024Review
- Gene Therapy for Retinal Degenerative Diseases: Progress, Challenges, and Future Directions.Investigative ophthalmology & visual science · 2023Review
- PAM-flexible Cas9-mediated base editing of a hemophilia B mutation in induced pluripotent stem cells.Communications medicine · 2023Article
- How to translate and implement the current science of gene therapy into haemophilia care?Therapeutic advances in hematology · 2023Review
- Therapeutic homology-independent targeted integration in retina and liver.Nature communications · 2022Article
- The Provocative Roles of Platelets in Liver Disease and Cancer.Frontiers in oncology · 2021Review
- A non-viral genome editing platform for site-specific insertion of large transgenes.Stem cell research & therapy · 2020Article
- Timing of Intensive Immunosuppression Impacts Risk of Transgene Antibodies after AAV Gene Therapy in Nonhuman Primates.Molecular therapy. Methods & clinical development · 2020Article
- Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy.Molecular therapy. Methods & clinical development · 2019Review
- Review
- Gene therapy for hemophilia: what does the future hold?Therapeutic advances in hematology · 2018Review
- Novel therapies and current clinical progress in hemophilia A.Therapeutic advances in hematology · 2018Review
- Emerging therapies for hemophilia: controversies and unanswered questions.F1000Research · 2018Review
- Review
- Seek and destroy: targeted adeno-associated viruses for gene delivery to hepatocellular carcinoma.Drug delivery · 2017Review
- Gene Therapy for Hemophilia.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Review
- [Construction of the pIRES2-ZsGreen1 eukaryotic expression vector of Factor Ⅸ gene and expression in HEK-293 cells].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2016Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
Abstract
introductionThe recent success of early-phase clinical trials for adeno-associated viral (AAV) liver-directed gene therapy for hemophilia B (HB) demonstrates the potential for gene therapy, in the future, to succeed protein-based prophylaxis therapy for HB. Significant obstacles, however, need to be overcome prior to widespread adoption. The largest obstacles include immune responses to the AAV capsid including preexisting neutralizing antibodies (NAbs) and a delayed cellular immune response. Emerging evidence suggests that the latter is vector-dose dependent. Furthermore, the development and eradication of inhibitors remains a significant safety concern. Similarly, biological differences between Factor VIII and Factor IX (FIX) impose challenges to direct adoption of the successes for HB to hemophilia A (HA). AREAS COVERED: The advantages and limitations of the current strategies addressing these obstacles for gene therapy for HB and HA are discussed, as well as vector manufacturing issues relevant to widespread adoption. Alternative strategies including both EXPERT OPINION: Our opinion is that these obstacles can be overcome with current approaches, and AAV-based gene therapy for HB will likely translate into future clinical care. Innovative approaches are, however, likely needed to solve the current problems obstructing HA gene therapy.
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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.