ReviewJournal of blood medicine2015
Recent trends in the gene therapy of β-thalassemia.
Review in Journal of blood medicine, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 37 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
37 citing papers in PubMed, 61 citations in OpenAlex.
- A Mass Spectrometry-Based Multiplexed Targeted Assay for Detection of Hemoglobinopathies from Dried Blood Spots.The Journal of molecular diagnostics : JMD · 2025Article
- Thalassemia in Sub-Saharan Africa: epidemiology, diagnosis, and management - a narrative review.Annals of medicine and surgery (2012) · 2025Review
- First clinical and pedigree study of rare HBB: c.316-90 A > G variant in β-globin gene in Chinese population using third-generation sequencing.Annals of hematology · 2025Article
- Upregulation of miR‑6747‑3p affects red blood cell lineage development and induces fetal hemoglobin expression by targeting BCL11A in β‑thalassemia.Molecular medicine reports · 2025Article
- Gene Therapy: A Revolutionary Step in Treating Thalassemia.Hematology reports · 2024Review
- Revolutionising health care: Exploring the latest advances in medical sciences.Journal of global health · 2023Article
- Health-related quality of life among thalassemia patients in Bangladesh using the SF-36 questionnaire.Scientific reports · 2023Article
- Combined approaches for increasing fetal hemoglobin (HbF) andFrontiers in genome editing · 2023Review
- The Roles of Mitophagy and Autophagy in Ineffective Erythropoiesis in β-Thalassemia.International journal of molecular sciences · 2022Review
- Current status of beta-thalassemia and its treatment strategies.Molecular genetics & genomic medicine · 2021Review
- Efficient CRISPR-Cas9-based genome editing of β-globin gene on erythroid cells from homozygous βMolecular therapy. Methods & clinical development · 2021Article
- Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34Scientific reports · 2020Article
- Inhibition of γ/β Globin Gene Switching in CD 34Indian journal of hematology & blood transfusion : an official journal of Indian Society of Hematology and Blood Transfusion · 2019Article
- In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.Scientific reports · 2019Article
- Omics Studies in Hemoglobinopathies.Molecular diagnosis & therapy · 2019Review
- MicroRNAs and Long Non-coding RNAs in Genetic Diseases.Molecular diagnosis & therapy · 2019Review
- Iron Toxicity and Hemopoietic Cell Transplantation: Time to Change the Paradigm.Mediterranean journal of hematology and infectious diseases · 2019Review
- Rapid and Sensitive Assessment of Globin Chains for Gene and Cell Therapy of Hemoglobinopathies.Human gene therapy methods · 2018Article
- BCL11A mRNA Targeting by miR-210: A Possible Network Regulating γ-Globin Gene Expression.International journal of molecular sciences · 2017Article
- Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
Corrections and comments
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Authors and funding
8 authors at 3 institutions in 3 countries.
Funding
Abstract
The β-thalassemias are a group of hereditary hematological diseases caused by over 300 mutations of the adult β-globin gene. Together with sickle cell anemia, thalassemia syndromes are among the most impactful diseases in developing countries, in which the lack of genetic counseling and prenatal diagnosis have contributed to the maintenance of a very high frequency of these genetic diseases in the population. Gene therapy for β-thalassemia has recently seen steadily accelerating progress and has reached a crossroads in its development. Presently, data from past and ongoing clinical trials guide the design of further clinical and preclinical studies based on gene augmentation, while fundamental insights into globin switching and new technology developments have inspired the investigation of novel gene-therapy approaches. Moreover, human erythropoietic stem cells from β-thalassemia patients have been the cellular targets of choice to date whereas future gene-therapy studies might increasingly draw on induced pluripotent stem cells. Herein, we summarize the most significant developments in β-thalassemia gene therapy over the last decade, with a strong emphasis on the most recent findings, for β-thalassemia model systems; for β-, γ-, and anti-sickling β-globin gene addition and combinatorial approaches including the latest results of clinical trials; and for novel approaches, such as transgene-mediated activation of γ-globin and genome editing using designer nucleases.
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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.