Current status of beta‐thalassemia and its treatment strategies
Background Thalassemia is an inherited hematological disorder categorized by a decrease
or absence of one or more of the globin chains synthesis. Beta‐thalassemia is caused by …
or absence of one or more of the globin chains synthesis. Beta‐thalassemia is caused by …
[HTML][HTML] Molecular basis of α-thalassemia
S Farashi, CL Harteveld - Blood Cells, Molecules, and Diseases, 2018 - Elsevier
Abstract α-Thalassemia is an inherited, autosomal recessive, disorder characterized by a
microcytic hypochromic anemia. It is one of the most common monogenic gene disorders in …
microcytic hypochromic anemia. It is one of the most common monogenic gene disorders in …
Comprehensive analysis and accurate quantification of unintended large gene modifications induced by CRISPR-Cas9 gene editing
Most genome editing analyses to date are based on quantifying small insertions and
deletions. Here, we show that CRISPR-Cas9 genome editing can induce large gene …
deletions. Here, we show that CRISPR-Cas9 genome editing can induce large gene …
[HTML][HTML] Molecular genetics of β-thalassemia: A narrative review
TH Jaing, TY Chang, SH Chen, CW Lin, YC Wen… - Medicine, 2021 - journals.lww.com
Abstract β-thalassemia is a hereditary hematological disease caused by over 350 mutations
in the β-globin gene (HBB). Identifying the genetic variants affecting fetal hemoglobin (HbF) …
in the β-globin gene (HBB). Identifying the genetic variants affecting fetal hemoglobin (HbF) …
Molecular basis and genetic modifiers of thalassemia
S Mettananda, DR Higgs - Hematology/Oncology Clinics, 2018 - hemonc.theclinics.com
Thalassemia is one of the most common monogenic disorders in the world. 1 It is estimated
that nearly 70,000 children with various forms of thalassemia are born each year. 2 …
that nearly 70,000 children with various forms of thalassemia are born each year. 2 …
Recent advances in CRISPR/Cas9-mediated knock-ins in mammalian cells
M Banan - Journal of biotechnology, 2020 - Elsevier
Since its inception, the CRISPR/Cas9 technology has been widely utilized for the targeted
insertion of donor DNAs into mammalian genomes. A shortcoming with the earlier knock-in …
insertion of donor DNAs into mammalian genomes. A shortcoming with the earlier knock-in …
Disrupting the adult globin promoter alleviates promoter competition and reactivates fetal globin gene expression
The benign condition hereditary persistence of fetal hemoglobin (HPFH) is known to
ameliorate symptoms of co-inherited β-hemoglobinopathies, such as sickle cell disease and …
ameliorate symptoms of co-inherited β-hemoglobinopathies, such as sickle cell disease and …
Recent progress in understanding and manipulating haemoglobin switching for the haemoglobinopathies
DS Vinjamur, DE Bauer… - British journal of …, 2018 - Wiley Online Library
The major β‐haemoglobinopathies, sickle cell disease and β‐thalassaemia, represent the
most common monogenic disorders worldwide and a steadily increasing global disease …
most common monogenic disorders worldwide and a steadily increasing global disease …
[PDF][PDF] Molecular basis and genetic modifiers of thalassemia
N Tesio, DE Bauer - Hematology/oncology clinics of North America, 2023 - Elsevier
Thalassemia syndromes are among the commonest monogenic disorders and represent a
substantial health burden worldwide. Here the authors have described globin genes …
substantial health burden worldwide. Here the authors have described globin genes …
[HTML][HTML] Molecular basis and diagnosis of thalassemia
JS Lee, S Im Cho, SS Park, MW Seong - Blood research, 2021 - synapse.koreamed.org
Thalassemia is characterized by the impaired synthesis of globin chains due to disease-
causing variants in α-or β-globin genes. In this review, we provide an overview of the …
causing variants in α-or β-globin genes. In this review, we provide an overview of the …