ArticleNature biotechnology2006
Stem cell-derived erythroid cells mediate long-term systemic protein delivery.
Article in Nature biotechnology, 2006. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 36 papers.
What it found
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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.
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Who cites it
36 citing papers in PubMed, 73 citations in OpenAlex.
- How Close Are We to Achieving Durable and Efficacious Gene Therapy for Hemophilia A and B?Genes · 2025Review
- The sound of silence: Transgene silencing in mammalian cell engineering.Cell systems · 2022Review
- Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins.Nature communications · 2020Article
- Adenovirus vectors in hematopoietic stem cell genome editing.FEBS letters · 2019Review
- High-level protein production in erythroid cells derived from in vivo transduced hematopoietic stem cells.Blood advances · 2019Article
- Functions of the Heterologous Intron-Derived Fragments Intra and Extra Factor IX-cDNA Coding Region on the Human Factor IX Expression in HepG2 and Hek-293T Cells.Iranian journal of biotechnology · 2018Article
- Article
- Sickle cells produce functional immune modulators and cytotoxics.American journal of hematology · 2017Article
- Bioengineering of differentiated hepatocytes with human factor IX-expressing plasmids in vitro.Bioengineered · 2016Article
- Lentiviral Vector-Mediated Correction of a Mouse Model of Leukocyte Adhesion Deficiency Type I.Human gene therapy · 2016Article
- De novo formed satellite DNA-based mammalian artificial chromosomes and their possible applications.Chromosome research : an international journal on the molecular, supramolecular and evolutionary aspects of chromosome biology · 2015Review
- Gene therapy for hemophilia.Frontiers in bioscience (Landmark edition) · 2015Review
- Development of Gene Transfer for Induction of Antigen-specific Tolerance.Molecular therapy. Methods & clinical development · 2014Article
- Platelet gene therapy by lentiviral gene delivery to hematopoietic stem cells restores hemostasis and induces humoral immune tolerance in FIX(null) mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- Concise review: stem cell-derived erythrocytes as upcoming players in blood transfusion.Stem cells (Dayton, Ohio) · 2012Review
- The potential of stem cells as an in vitro source of red blood cells for transfusion.Cell stem cell · 2012Review
- Gene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation.British journal of haematology · 2012Review
- Animal models of hemophilia.Progress in molecular biology and translational science · 2012Review
- Cell- and gene-based therapeutic approaches for neurological deficits in mucopolysaccharidoses.Current pharmaceutical biotechnology · 2011Review
- Factor VIII delivered by haematopoietic stem cell-derived B cells corrects the phenotype of haemophilia A mice.Thrombosis and haemostasis · 2011Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 1 institution in 1 country.
Funding
Abstract
We demonstrate here the capacity of erythroid cells to mediate long-term, systemic and therapeutic protein delivery in vivo. By targeting human factor IX (hFIX) expression to late-stage erythropoiesis, we achieve long-term hFIX secretion at levels significantly higher (>tenfold) than those obtained with an archetypal ubiquitous promoter in a mouse model of hemophilia B. Erythroid cell-derived hFIX is biologically active, resulting in phenotypic correction of the bleeding disorder. In addition to achieving high expression levels and resistance to transcriptional silencing, red cell-mediated protein delivery offers multiple advantages including immune tolerance induction, reduction of the risk of insertional oncogenesis and relative ease of application by either engrafting transduced hematopoietic stem cells or transfusing ex vivo-generated, stem cell-derived erythroid cells.
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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.