Results 91 to 100 of about 51,483 (212)
Granulocyte\u2013colony stimulating factor plus plerixafor inpatients with \u2013thalassemia major results in the effective mobilization of primitiveCD34+ cells with specific gene expression profile [PDF]
, 2017 Successful gene therapy for \u3b2-thalassemia requires optimal numbers of autologous gene-transduced hematopoietic stem and progenitor cells (HSPCs) with high repopulating capacity.
Aldo, F. +9 more
core +2 more sources
European Journal of Haematology, EarlyView.ABSTRACT In the phase 2, double‐blind, randomized controlled BEYOND trial (NCT03342404), luspatercept increased hemoglobin levels in patients with non‐transfusion‐dependent β‐thalassemia (NTDT). This study assessed long‐term effects of luspatercept on patient‐reported outcomes (PROs), using data from BEYOND and patients who continued luspatercept ...
Khaled M. Musallam +12 more
wiley +1 more source
Haematologica, 2009 Ten patients with thalassemia intermedia with variable severity and apparent simple heterozygosis for β0 39 C>T nonsense mutation were submitted to clinical, hematologic and molecular studies.
Maria Carla Sollaino +5 more
doaj +1 more source
Quantitative analysis of DNA‐GATA1 binding alterations linked to hematopoietic disorders
The FEBS Journal, EarlyView.Native holdup allows the quantitative determination of affinities between full‐length transcription factors and DNA. Mutations in either the protein or the DNA can modulate binding strength, which can be precisely quantified using this approach. Applied to GATA1, it revealed mutations that alter DNA binding.
Boglarka Zambo +6 more
wiley +1 more source
Third-degree heart block in thalassemia major: A case report [PDF]
, 2012 Background: First and second-degree heart blocks are partly common rhythm disorders in thalassemic patients but complete heart block is a very rare complication of iron overload cardiomyopathy.
Hosseini, S.M., Maleki, A.R., Nikyar, B.
core +1 more source
Tropical Medicine &International Health, EarlyView.ABSTRACT Introduction Poor glycaemic control, indicated by a haemoglobin A1c (HbA1c) level of 7% or higher, increases the risk of active tuberculosis (TB) in patients with diabetes, while active TB can worsen glucose tolerance and insulin resistance, contributing to type‐2 diabetes mellitus (DM) progression.
Augustine Asare Boadu +11 more
wiley +1 more source
Intervention and Prevention of Hereditary Hemolytic Disorders in Two Ethnic Communities of Sundargarh District of Orissa, India: An Experience from KAP Studies [PDF]
, 2010 Hereditary hemolytic disorders are important public health challenges in India. They cause a high degree of morbidity, mortality and fetal wastage in vulnerable communities.
Balgir, RS
core +1 more source
Current Progress in Therapeutic Gene Editing for Monogenic Diseases [PDF]
, 2016 Programmable nucleases allow defined alterations in the genome with ease-of-use, efficiency, and specificity. Their availability has led to accurate and widespread genome engineering, with multiple applications in basic research, biotechnology, and ...
Moore, Marc +2 more
core +1 more source
Pediatric Blood &Cancer, Volume 73, Issue 5, May 2026.ABSTRACT Hemoglobinopathies are prevalent globally; diagnosis is complex in high genetic admixture populations like Brazil. We report, in two pediatric siblings, the first documented cases in Brazil of heterozygosity for hemoglobin (Hb) O‐Arab with coinheritance of α‐thalassemia (αα/−α4.2; −α3.7/−α4.2), resulting in microcytic and hypochromic anemia ...
Elisângela de Souza Miranda Muynarsk +9 more
wiley +1 more source
PBI22 Rethinking the Health-Economic Evaluation Framework for GENE Therapies: The Betibeglogene Autotemcel (BETI-CEL) Case in Î’-Thalassemia [PDF]
Value in Health, 2020 L. Undreiner +3 more
openaire +1 more source