Results 11 to 20 of about 38,246 (331)
International Journal of Neonatal Screening, 2018 Sickle cell disease (SCD) and other hemoglobinopathies are a major health concern with a high burden of disease worldwide. Since the implementation of newborn screening (NBS) for SCD and other hemoglobinopathies in several regions of the world, technical
Claudia Frömmel
exaly +4 more sources
A Small Key for a Heavy Door: Genetic Therapies for the Treatment of Hemoglobinopathies [PDF]
Frontiers in Genome Editing, 2021 Throughout the past decades, the search for a treatment for severe hemoglobinopathies has gained increased interest within the scientific community.
Hidde A. Zittersteijn +6 more
openalex +2 more sources
The hemoglobinopathies, molecular disease mechanisms and diagnostics
International Journal of Laboratory Hematology, 2022 Hemoglobinopathies are the most common monogenic disorders in the world with an ever increasing global disease burden each year. As most hemoglobinopathies show recessive inheritance carriers are usually clinically silent.
Cornelis L Harteveld
exaly +2 more sources
Asian Journal of Transfusion Science, 2022 CONTEXT: Hemoglobinopathies are the most common heterogeneous group of monogenetic disorder in the world and its prevalence varies with geographical regions.
Virender Singh +4 more
doaj +2 more sources
Genome Editing for β-Hemoglobinopathies: Advances and Challenges
Journal of Clinical Medicine, 2021 β-hemoglobinopathies are the most common genetic disorders worldwide and are caused by mutations affecting the production or the structure of adult hemoglobin.
Giacomo Frati, Annarita Miccio
exaly +2 more sources
Gene therapy of hemoglobinopathies: progress and future challenges
Human Molecular Genetics, 2019 Recently, gene therapy clinical trials have been successfully applied to hemoglobinopathies, such as sickle cell disease (SCD) and β-thalassemia. Among the great discoveries that led to the design of genetic approaches to cure these disorders is the ...
Annarita Miccio +2 more
exaly +2 more sources
Saudi Medical Journal, 2021 Hereditary hemoglobin disorders with thalassemia and sickle-cell anemia are the most common monogenic diseases in the world. It is estimated that about 1-5% of the global population is the carriers of a genetic thalassemia mutation. Hemoglobinopathies are among the most common hereditary blood diseases also in Turkey and are an important health problem
Arpacı, Abdullah +2 more
openaire +3 more sources
Hemoglobinopathies in Iran: An Updated Review
International Journal of Hematology-Oncology and Stem Cell Research, 2020 Hemoglobinopathies are the most common single gene disorders (monogenic disorders) in the world population. Due to specific position of Iran and the presence of multi-ethnic groups in the country, there are many varieties in the molecular genetics and ...
Abolfazl Nasiri +2 more
doaj +2 more sources
Molecular studies reveal a concordant KEL genotyping between patients with hemoglobinopathies and blood donors in São Paulo City, Brazil [PDF]
, 2008 Universidade Federal de São Paulo, Discipline Hematol & Transfus Med, BR-04023092 São Paulo, BrazilUniversidade Federal de São Paulo, Discipline Hematol & Transfus Med, BR-04023092 São Paulo, BrazilWeb of ...
Bordin, Jose Orlando +4 more
core +3 more sources
Transfusion strategies in hemoglobinopathies: what the latest update of Good Practices tells us in the era of new therapeutic advances. [PDF]
Blood TransfusSchieppati F.
europepmc +3 more sources