Analysis of Common Beta-Thalassemia (β-Thalassemia) Mutations in East Java, Indonesia.
PMID:35911837
Exploring the crosstalk between long non-coding RNAs and microRNAs to unravel potential prognostic and therapeutic biomarkers in β-thalassemia.
PMID:35717472
Global Globin Network Consensus Paper: Classification and Stratified Roadmaps for Improved Thalassaemia Care and Prevention in 32 Countries.
PMID:35455667
Prevalence of transfusion-transmitted infections in multiple blood transfusion-dependent thalassemic patients in Asia: A systemic review.
PMID:35452334
Plasma proteome profiling combined with clinical and genetic features reveals the pathophysiological characteristics of β-thalassemia.
PMID:35378860
Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial.
PMID:34980909
The EHA Research Roadmap: Normal Hematopoiesis.
PMID:34853826
Molecular genetics of β-thalassemia: A narrative review.
PMID:34766559
Evaluation of β-Thalassaemia Cases for Common Mutations in Western Rajasthan.
PMID:34744352
A Small Key for a Heavy Door: Genetic Therapies for the Treatment of Hemoglobinopathies.
PMID:34713239
β-Hemoglobinopathies: The Test Bench for Genome Editing-Based Therapeutic Strategies.
PMID:34713219
Quality of Life and Related Paraclinical Factors in Iranian Patients with Transfusion-Dependent Thalassemia.
PMID:34457009
Therapy Development by Genome Editing of Hematopoietic Stem Cells.
PMID:34198536
Systematic evaluation of the effect of polyadenylation signal variants on the expression of disease-associated genes.
PMID:33875481
Physiological and Aberrant γ-Globin Transcription During Development.
PMID:33869190
Genome editing using CRISPR/Cas9 to treat hereditary hematological disorders.
PMID:33750926
A study of red blood cell alloimmunization and autoimmunization among 200 multitransfused Egyptian β thalassemia patients.
PMID:33273689
Relative and Absolute Quantification of Aberrant and Normal Splice Variants in HBBIVSI-110 (G > A) β-Thalassemia.
PMID:32933098
CRISPR-mediated gene modification of hematopoietic stem cells with beta-thalassemia IVS-1-110 mutation.
PMID:32912325
Changing patterns in the epidemiology of β-thalassemia.
PMID:32886826
Effect of Mutations on mRNA and Globin Stability: The Cases of Hb Bernalda/Groene Hart and Hb Southern Italy.
PMID:32751969
Effect of HBB genotype on survival in a cohort of transfusion-dependent thalassemia patients in Cyprus.
PMID:32732363
Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34+ HSPCs by CRISPR/Cas9 for the induction of fetal hemoglobin.
PMID:32576837
Care-Related Quality of Life of Caregivers of Beta-Thalassemia Major Children: An Epidemiological Study in Eastern India.
PMID:32538034
β-Thalassemia pathogenic variants in a cohort of children from the East African coast.
PMID:32394645
The prevalence of hepatitis C virus infection in β-thalassemia patients in Pakistan: a systematic review and meta-analysis.
PMID:32349737
Hb S/β-Thalassemia in the REDS-III Brazil Sickle Cell Disease Cohort: Clinical, Laboratory and Molecular Characteristics.
PMID:32172616
Identification of a novel pre-terminating mutation in human HBB gene as a cause of β0-thalassemia phenotype.
PMID:31934147
The Scope for Thalassemia Gene Therapy by Disruption of Aberrant Regulatory Elements.
PMID:31766235
Genetic Modifiers at the Crossroads of Personalised Medicine for Haemoglobinopathies.
PMID:31717530
Improved efficiency and patient safety through bespoke electronic thalassaemia care module.
PMID:31533923
Family planning practices in couples with children affected by β-thalassemia major and its relationship with their education: An epidemiological study.
PMID:31463328
LOVD-DASH: A comprehensive LOVD database coupled with diagnosis and an at-risk assessment system for hemoglobinopathies.
PMID:31286593
A novel mutation in the erythroid transcription factor KLF1 is likely responsible for ameliorating β-thalassemia major.
PMID:31115947
Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas-and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells.
PMID:31004018
Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases.
PMID:30945166
Editing aberrant splice sites efficiently restores β-globin expression in β-thalassemia.
PMID:30704988
Short-hairpin RNA against aberrant HBBIVSI-110(G>A) mRNA restores β-globin levels in a novel cell model and acts as mono- and combination therapy for β-thalassemia in primary hematopoietic stem cells.
PMID:29700171
Molecular basis of β thalassemia and potential therapeutic targets.
PMID:28651846
Two novel C-terminal frameshift mutations in the β-globin gene lead to rapid mRNA decay.
PMID:28595636
β-Thalassemia Distribution in the Old World: an Ancient Disease Seen from a Historical Standpoint.
PMID:28293406
The molecular spectrum and distribution of haemoglobinopathies in Cyprus: a 20-year retrospective study.
PMID:27199182
The European Hematology Association Roadmap for European Hematology Research: a consensus document.
PMID:26819058
Recent trends in the gene therapy of β-thalassemia.
PMID:25737641