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Thalassemia (severe form)
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Publications and studies (1520)
- The development of an advanced lentiviral gene therapy for beta-thalassemia. (2025/12/31) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. The second reported case of a hemolytic transfusion reaction caused by anti-Sc2: a clinical diagnosis. (2025/12/31) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Ineffective Erythropoiesis Markers in β-Thalassemia: A Systematic Review. (2025/12/31) ♡
- Association of IL28B Polymorphisms With Hepatitis C Susceptibility in Southern Iran's β-Thalassemia Population: A Cross-Sectional Study. (2025/12/27) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Advances in Gene Therapy for Inherited Haemoglobinopathies. (2025/12/27) ♡
- Functional Abnormalities of the Endocrine System in Beta-Thalassemia Major Patients: Insights From a Hospital-Based Observational Study. (2025/12/27) ♡
- Nutritional deficiencies among adults with beta thalassemia major in Vietnam. (2025/12/26) ♡
- A New Method for Screening Thalassemia Patients Using Mid-Infrared Spectroscopy. (2025/12/24) ♡
- Carrier screening for multiple complex monogenic diseases using long-read sequencing: a population-based study of premarital couples in Shanghai. (2025/12/22) ♡
- CRISPR editing of HPFH3 genotype induces γ-globin expression and reverses sickle cell disease and β-thalassemia phenotypes. (2025/12/21) ♡
- Lowering cyclosporine trough concentration threshold effectively mitigates acute graft-versus-host disease in children with severe β-Thalassemia post hematopoietic stem cell transplantation. (2025/12/19) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Comprehensive genetic screening of in vitro fertilized embryos using preimplantation genetic testing for monogenic gene disorders via the Sanger sequencing technique. (2025/12/18) ♡
- Novel LRF/ZBTB7A variants and known HbF-modulating SNPs in transfusion-dependent β-thalassemia. (2025/12/18) ♡
- Effects of fluconazole and voriconazole on cyclosporine levels and toxicity in allogenic hematopoietic stem cell transplant recipients: A comprehensive analysis. (2025/12/17) ♡
- Alterations in iron status predict cardiac response to blood transfusion in β-thalassemia major. (2025/12/17) ♡
- Fragmented QRS duration as a marker of cardiac and hepatic iron overload: a cross-sectional study. (2025/12/17) ♡
- Endocrine complications in patients with β-thalassemia major receiving iron-chelation therapy. (2025/12/17) ♡
- Analysis of Hematological Parameters in Relation to Genotypes in 497 Patients with Hemoglobin H Disease. (2025/12/16) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Therapeutic applications of CRISPR-Cas9 gene editing. (2025/12/16) ♡
- The willingness and perceptions of Surinamese individuals in the Netherlands on living tissue donation: A qualitative and exploratory study. (2025/12/15) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Gene Therapy of Beta Hemoglobinopathies. (2025/12/15) ♡
- Family trios/quartets analysis based on the Newborn Genomic Atlas for Thalassemia project in Guangxi. (2025/12/12) ♡
- Iron-associated central macular ganglion cell complex thinning and choroidal vascularity index elevation in transfusion-dependent β-thalassemia: potential OCT/OCTA biomarkers. (2025/12/12) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Beyond the Exome: The Role of Noncoding and Regulatory Variants in Monogenic Diseases. (2025/12/12) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Changes in attitudes towards prenatal diagnosis for haemoglobinopathies: a 40-year retrospective observational study in Northern Italy. (2025/12/07) ♡
- Downregulation of ZBTB7A/LRF increases fetal hemoglobin expression in β(0)-thalassemia/hemoglobin E erythroid cells. (2025/12/05) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Thalassemia and hypercoagulability. (2025/12/05) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. A Rare Combination of Hereditary Folate Malabsorption (SLC46A1 Gene Variant) and Beta-Thalassemia Trait. (2025/12/05) ♡
- Liver Tissue Mapping in Transfusion-Dependent β-Thalassemia: Reproducibility and Clinical Insights from Multiparametric MRI. (2025/12/04) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Rare variant hemoglobin Köln in Southern India—an underdiagnosed entity? (2025/12/03) ♡
- Mixed vaginal infection status in women infected with Trichomonas vaginalis: comparison of microscopy method and metagenomic sequencing analysis. (2025/12/03) ♡
- MAZ regulates fetal hemoglobin repression by activating MYB transcription. (2025/12/02) ♡
- Deferiprone therapy improves the oxidative status of LDL in patients with β-thalassaemia/HbE. (2025/12/02) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Host Immunogenetics and Chronic HCV Infection Shape Atopic Risk in Pediatric Beta-Thalassemia: A Genotype-Phenotype Study. (2025/12/02) ♡
- IGHG4: innovative diagnostic biomarkers for iron overload in β-thalassemia patients. (2025/12/01) ♡
- Association of healthy eating behaviours and comorbidity on quality of life among adults with beta-thalassemia major: a moderation analysis. (2025/12/01) ♡
- A pilot study on the prevalence and patterns of haemoglobinopathies in Datia District, Madhya Pradesh, India. (2025/12/01) ♡
- Genetic mutations of Transfusion Dependent Thalassaemia (TDT) in paediatric patients. (2025/12/01) ♡
- Haematological indices as screening tool in distinguishing beta thalassemia trait and iron deficiency anaemia: Insights from a tertiary hospital study. (2025/12/01) ♡
- [Screening Results of Thalassemia and Analysis of Rare Genotypes]. (2025/12/01) ♡
- [Analysis of Gene Types and Clinical Characteristics of Thalassemia in Children in Nanchong Area]. (2025/12/01) ♡
- [The Role of MiR-709 in Erythroid Development and Its Correlation with Multiple Hematological Diseases]. (2025/12/01) ♡
- Association of CYP2C19 gene single nucleotide polymorphisms (rs12248560 and rs4244285) with response to thalidomide in transfusion dependent β-thalassemia patients- a 12-months follow-up study. (2025/12/01) ♡
- UGT1A6 variants and deferiprone-induced ADRs: a complication-specific analysis in Iranian thalassemia patients. (2025/12/01) ♡
- Determinants of intertransfusion interval in children with transfusion-dependent thalassemia: a retrospective single-center cohort study in China. (2025/11/30) ♡
- Assessment of Adherence to Iron Chelation Therapy Among Thalassemia Patients in Palestine. (2025/11/29) ♡
- Epidemiology and economic burden of selected rare genetic diseases in Germany - a claims database study. (2025/11/28) ♡
- Base editing of β(0)-thalassemia mutations as a therapeutic strategy for severe β-hemoglobinopathies. (2025/11/26) ♡
- Randomized researchiParticipants were divided into groups by lottery and compared with each other. This reduces the chance that a difference is due to something other than the treatment. Safety and pharmacodynamics of the ferroportin inhibitor vamifeport in patients with non-transfusion-dependent β-thalassemia: results from a randomized phase 2a study. (2025/11/25) ♡
- Deep Vein Thrombosis in a Patient With Thalassemia Minor: A Case Report. (2025/11/23) ♡
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