ReviewBlood2013
Development of gene therapy for blood disorders: an update.
Review in Blood, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
19 citing papers in PubMed, 52 citations in OpenAlex.
- Viral-based gene therapy clinical trials for immune deficiencies and blood disorders from 2013 until 2023 - an overview.Regenerative therapy · 2025Review
- Engineered and hybrid human megakaryocytic extracellular vesicles for targeted non-viral cargo delivery to hematopoietic (blood) stem and progenitor cells.Frontiers in bioengineering and biotechnology · 2024Article
- Megakaryocyte membrane-wrapped nanoparticles for targeted cargo delivery to hematopoietic stem and progenitor cells.Bioengineering & translational medicine · 2023Article
- Novel genetic therapeutic approaches for modulating the severity of β-thalassemia (Review).Biomedical reports · 2020Review
- Therapies for rare diseases: therapeutic modalities, progress and challenges ahead.Nature reviews. Drug discovery · 2020Review
- Transfection by cationic gemini lipids and surfactants.MedChemComm · 2018Review
- Short-course rapamycin treatment enables engraftment of immunogenic gene-engineered bone marrow under low-dose irradiation to permit long-term immunological tolerance.Stem cell research & therapy · 2017Article
- Lentiviral Transfer of γ-Globin with Fusion Gene NUP98-HOXA10HD Expands Hematopoietic Stem Cells and Ameliorates Murine β-Thalassemia.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
- Cure for thalassemia major - from allogeneic hematopoietic stem cell transplantation to gene therapy.Haematologica · 2017Review
- CRISPR-Cas9 technology and its application in haematological disorders.British journal of haematology · 2016Review
- Review
- Functional Restoration of gp91phox-Oxidase Activity by BAC Transgenesis and Gene Targeting in X-linked Chronic Granulomatous Disease iPSCs.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Article
- Hepatocyte Heparan Sulfate Is Required for Adeno-Associated Virus 2 but Dispensable for Adenovirus 5 Liver Transduction In Vivo.Journal of virology · 2016Article
- Targeted Gene Correction in Osteopetrotic-Induced Pluripotent Stem Cells for the Generation of Functional Osteoclasts.Stem cell reports · 2015Article
- High-level production of replication-defective human immunodeficiency type 1 virus vector particles using helper-dependent adenovirus vectors.Molecular therapy. Methods & clinical development · 2015Article
- Therapeutic applications of extracellular vesicles: clinical promise and open questions.Annual review of pharmacology and toxicology · 2015Review
- State-of-the-art human gene therapy: part II. Gene therapy strategies and clinical applications.Discovery medicine · 2014Review
- Pseudotyped murine leukemia virus for schistosome transgenesis: approaches, methods and perspectives.Transgenic research · 2014Article
- Non-integrating gamma-retroviral vectors as a versatile tool for transient zinc-finger nuclease delivery.Scientific reports · 2014Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
Abstract
This review addresses the current status of gene therapy for immunodeficiencies, chronic granulomatous disease, suicide gene therapy for graft-versus-host disease, viral infections, malignant hematologic disorders, hemophilia, and the hemoglobin disorders. New developments in vector design have fostered improved expression as well as enhanced safety, particularly of integrating retroviral vectors. Several immunodeficiencies have been treated successfully by stem cell-targeted, retroviral-mediated gene transfer with reconstitution of the immune system following infusion of the transduced cells. In a trial for hemophilia B, long-term expression of human FIX has been observed following adeno-associated viral vector-mediated gene transfer into the liver. This approach should be successful in treating any disorder in which liver production of a specific protein is therapeutic.
Indexed as
Identifiers
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.