ReviewHaemophilia : the official journal of the World Federation of Hemophilia2021
WFH State-of-the-art paper 2020: In vivo lentiviral vector gene therapy for haemophilia.
Review in Haemophilia : the official journal of the World Federation of Hemophilia, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
What it found
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Who cites it
16 citing papers in PubMed, 28 citations in OpenAlex.
- Long-Term Liver-Targeted AAV8 Gene Therapy for Mucopolysaccharidosis IVA.Current issues in molecular biology · 2025Article
- Article
- GP64-pseudotyped lentiviral vectors target liver endothelial cells and correct hemophilia A mice.EMBO molecular medicine · 2024Article
- Non-viral and viral delivery systems for hemophilia A therapy: recent development and prospects.Annals of hematology · 2024Review
- Hemophilia Healing with AAV: Navigating the Frontier of Gene Therapy.Current gene therapy · 2024Review
- Therapeutic potential of fetal liver cell transplantation in hemophilia A mice.Haematologica · 2023Article
- Review
- Characteristics of BAY 2599023 in the Current Treatment Landscape of Hemophilia A Gene Therapy.Current gene therapy · 2023Review
- Gene transfer and genome editing for familial hypercholesterolemia.Frontiers in molecular medicine · 2023Review
- Gene therapy for hemophilia: looking beyond factor expression.Experimental biology and medicine (Maywood, N.J.) · 2022Review
- Impact of novel hemophilia therapies around the world.Research and practice in thrombosis and haemostasis · 2022Article
- Gene Therapy in Hemophilia: Recent Advances.International journal of molecular sciences · 2021Review
- Non-genotoxic conditioning facilitates hematopoietic stem cell gene therapy for hemophilia A using bioengineered factor VIII.Molecular therapy. Methods & clinical development · 2021Article
- Hemophilia Gene Therapy: Approaching the First Licensed Product.HemaSphere · 2021Review
- WFH State-of-the-art paper 2020: In vivo lentiviral vector gene therapy for haemophilia.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Review
- Review
Corrections and comments
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Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Over the last decade, the development of new treatments for haemophilia has progressed at a very rapid pace. Despite all the promising advances in protein products, the prospect offered by gene therapy of a single potentially lifelong treatment remains attractive for people with haemophilia. Transfer to the liver of coagulation factor VIII (FVIII) or factor IX (FIX) transgenes has indeed the potential to stably restore the dysfunctional coagulation process. Recombinant adeno-associated virus (AAV)-derived vectors are widely employed for liver-directed gene therapy, given their very good efficacy and safety profile, shown in several preclinical and clinical studies. However, there are some limitations associated with AAV vectors, such as their predominantly episomal nature in the nucleus of target cells and the widespread pre-existing immunity against the parental virus in humans. By contrast, HIV-derived lentiviral vectors (LV) integrate into the target cell chromatin and are maintained as the cells duplicate their genome, a potential advantage for establishing long-term expression especially in paediatric patients, in which the liver undergoes substantial growth. Systemic administration of LV allowed stable multi-year transgene expression in the liver of mice and dogs. More recently, improved phagocytosis-shielded LV were generated, which, following intravenous administration to non-human primates, showed selective targeting of liver and spleen and enhanced hepatocyte gene transfer, achieving up to supra-normal activity of both human FVIII and FIX transgenes. These studies support further preclinical assessment and clinical evaluation of in vivo liver-directed LV gene therapy for haemophilia.
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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.