Results 11 to 20 of about 1,827,024 (396)
New England Journal of Medicine, 2017 Sickle cell disease is an increasing global health problem. Estimates suggest that every year approximately 300,000 infants are born with sickle cell anemia, which is defined as homozygosity for the sickle hemoglobin (HbS) gene (i.e., for a missense ...
F. Piel, M. Steinberg, D. Rees
semanticscholar +16 more sources
Nature Reviews Disease Primers, 2018 Sickle cell disease (SCD) is a group of inherited disorders caused by mutations in HBB, which encodes haemoglobin subunit β. The incidence is estimated to be between 300,000 and 400,000 neonates globally each year, the majority in sub-Saharan Africa. Haemoglobin molecules that include mutant sickle β-globin subunits can polymerize; erythrocytes that ...
G. Kato +10 more
semanticscholar +11 more sources
Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease.
New England Journal of Medicine, 2021 BACKGROUND Sickle cell disease is characterized by the painful recurrence of vaso-occlusive events. Gene therapy with the use of LentiGlobin for sickle cell disease (bb1111; lovotibeglogene autotemcel) consists of autologous transplantation of ...
J. Kanter +18 more
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Frontiers in Medicine, 2021 Patients with sickle cell disease often undergo frequent blood transfusions. This increases their exposure to red blood cell alloantigens of donor units, thus making it more likely that they produce alloantibodies.
Salomé Conrath +6 more
doaj +1 more source
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
Acute Myeloid Leukemia Case after Gene Therapy for Sickle Cell Disease.
New England Journal of Medicine, 2021 Gene therapy with LentiGlobin for sickle cell disease (bb1111, lovotibeglogene autotemcel) consists of autologous transplantation of a patient's hematopoietic stem cells transduced with the BB305 lentiviral vector that encodes the βA-T87Q-globin gene ...
S. Goyal +16 more
semanticscholar +1 more source
The Lancet, 2010 Sickle-cell disease is one of the most common severe monogenic disorders in the world. Haemoglobin polymerisation, leading to erythrocyte rigidity and vaso-occlusion, is central to the pathophysiology of this disease, although the importance of chronic anaemia, haemolysis, and vasculopathy has been established.
Rees, David C. +2 more
openaire +7 more sources
American Society of Hematology 2020 guidelines for sickle cell disease: transfusion support.
Blood Advances, 2020 BACKGROUND Red cell transfusions remain a mainstay of therapy for patients with sickle cell disease (SCD), but pose significant clinical challenges. Guidance for specific indications and administration of transfusion, as well as screening, prevention ...
S. Chou +14 more
semanticscholar +1 more source
Biomedicines, 2022 The safety profile of hydroxyurea (HU) in patients with sickle-cell disease (SCD) is relatively well known. However, despite the suspected association of HU with myeloid neoplasms in myeloproliferative neoplasms (MPN), and the publication of sporadic ...
Pagona Flevari +10 more
doaj +1 more source