Results 41 to 50 of about 398,944 (237)
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
New England Journal of Medicine, 2020 Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is a transcription factor that represses γ-globin expression and fetal hemoglobin in
H. Frangoul +25 more
semanticscholar +1 more source
Advances in the diagnosis and treatment of sickle cell disease
Journal of Hematology & Oncology, 2022 Sickle cell disease (SCD), which affects approximately 100,000 individuals in the USA and more than 3 million worldwide, is caused by mutations in the βb globin gene that result in sickle hemoglobin production.
A. Brandow, R. Liem
semanticscholar +1 more source
Prevalence of hemoglobin abnormalities in an apparently healthy population in Benin
Hematology, Transfusion and Cell Therapy, 2020 Background: Sickle cell disease is the most common monogenic disorder in humans and is a major public health concern in sub-Saharan Africa. In Benin, the prevalence of sickle cell disease is estimated to be 4.8%.
Alban Zohoun +3 more
doaj +1 more source
The Evolving Pharmacotherapeutic Landscape for the Treatment of Sickle Cell Disease. [PDF]
, 2020 Sickle cell disease (SCD) is an extremely heterogeneous disease that has been associated with global morbidity and early mortality. More effective and inexpensive therapies are needed.
Ballas, Samir K
core +1 more source
Comorbidities in aging patients with sickle cell disease. [PDF]
, 2018 Sickle cell disease (SCD) in general and sickle cell anemia in particular is a highly complex disorder both at the molecular and clinical levels. Although the molecular lesion is a single-point mutation, the sickle gene is pleiotropic in nature causing ...
Ballas, Samir K.
core +2 more sources
Nutritional status of young children with inherited blood disorders in western Kenya. [PDF]
, 2014 To determine the association between a range of inherited blood disorders and indicators of poor nutrition, we analyzed data from a population-based, cross-sectional survey of 882 children 6–35 months of age in western Kenya.
Ruth, LJ +4 more
core +1 more source
Revista Brasileira de Hematologia e Hemoterapia, 2012 BACKGROUND: Sickle cell disease is a hemoglobinopathy characterized by hemolytic anemia, increased susceptibility to infections and recurrent vaso-occlusive crises that reduces the quality of life of sufferers.
Darcielle Bruna Dias Elias +5 more
doaj +1 more source
Haemoglobinopathies and newborn haemoglobinopathy screening in Germany. [PDF]
, 2009 Germany has been an immigration country since the early 1950s. In December 2007, 6.7 million non-German citizens lived in the country. However, the total number of citizens with a migration background is 15–20 million, about 9 million of whom come ...
Dickerhoff, R. +2 more
core +1 more source
Kidney Disease among Patients with Sickle Cell Disease, Hemoglobin SS and SC.
American Society of Nephrology. Clinical Journal, 2016 BACKGROUND AND OBJECTIVES Sickle cell disease (SCD) is an inherited anemia that afflicts millions worldwide. Kidney disease is a major contributor to its morbidity and mortality.
P. Drawz +7 more
semanticscholar +1 more source
The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells. [PDF]
, 2015 Lentiviral vectors designed for the treatment of the hemoglobinopathies require the inclusion of regulatory and strong enhancer elements to achieve sufficient expression of the β-globin transgene.
Baldwin, Kismet M +12 more
core +8 more sources