Results 11 to 20 of about 10,746,438 (360)

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, J. Tisdale, M. Schmidt, J. Kanter, J. Jaroscak, Dustin Whitney, H. Bitter, P. Gregory, G. Parsons, Marianna Foos, Ashish S. Yeri, Maple Gioia, S. Voytek, Alexandra L. Miller, J. Lynch, R. Colvin, M. Bonner   +16 more
semanticscholar   +1 more source

Sickle cell disease patients with COVID‐19 in Guadeloupe: Surprisingly favorable outcomes

eJHaem, 2022
We investigate risk factors for hospitalization and difference between sickle cell syndromes in a cohort of COVID‐19 sickle cell disease (SCD) adult patients managed in the Reference Center of Guadeloupe.
Emmanuelle Bernit, Marc Romana, Scylia Alexis‐Fardini, Vanessa Tarer, Pierre‐Marie Roger, Lydia Doumdo, Eléonore Petras, Corine Charneau, Benoit Tressières, Marie Dominique Hardy Dessources, Maryse Etienne‐Julan   +10 more
doaj   +1 more source

Techniques for the Detection of Sickle Cell Disease: A Review

Micromachines, 2021
Sickle cell disease (SCD) is a widespread disease caused by a mutation in the beta-globin gene that leads to the production of abnormal hemoglobin called hemoglobin S.
W. Arishi, H. Alhadrami, M. Zourob
semanticscholar   +1 more source

Treatment of sickle cell disease by increasing oxygen affinity of hemoglobin.

Blood, 2021
The issue of treating sickle cell disease with drugs that increase hemoglobin oxygen affinity has come to the fore with the FDA approval in 2019 of voxelotor, the only anti-sickling drug approved since hydroxyurea in 1998.
E. Henry, B. Metaferia, Quan Li, Julia Harper, R. Best, Kristen E. Glass, Troy Cellmer, Emily B. Dunkelberger, Anna K. Conrey, S. Thein, H. Bunn, W. Eaton   +11 more
semanticscholar   +1 more source

Red cell transfusion and alloimmunization in sickle cell disease

Haematologica, 2021
Red cell transfusion remains a critical component of care for acute and chronic complications of sickle cell disease. Randomized clinical trials demonstrated the benefits of transfusion therapy for prevention of primary and secondary strokes and ...
G. Linder, S. Chou
semanticscholar   +1 more source

Patterns and patient factors associated with loss to follow-up in the Muhimbili sickle cell cohort, Tanzania

BMC Health Services Research, 2020
Background Monitoring patient’s clinical attendance is a crucial means of improving retention in care and treatment programmes. Sickle cell patients’ outcomes are improved by participation in comprehensive care programmes, but these benefits cannot be ...
Upendo Masamu, Raphael Z. Sangeda, Daniel Kandonga, Jesca Ondengo, Flora Ndobho, Bruno Mmbando, Siana Nkya, Khadija Msami, Julie Makani   +8 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, D. Altshuler, M. Cappellini, Yi-Shan Chen, J. Domm, Brenda K. Eustace, J. Foell, J. de la Fuente, S. Grupp, R. Handgretinger, Tony W. Ho, A. Kattamis, A. Kernytsky, J. Lekstrom-Himes, Amanda M Li, F. Locatelli, M. Mapara, M. de Montalembert, D. Rondelli, Akshay Sharma, S. Sheth, S. Soni, M. Steinberg, D. Wall, Angela Yen, S. Corbacioglu   +25 more
semanticscholar   +1 more source

Sickle-cell disease

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., Williams, Thomas N., Gladwin, Mark T.   +2 more
openaire   +6 more sources

HIV-1 Tat phosphorylation on Ser-16 residue modulates HIV-1 transcription

Retrovirology, 2018
Background HIV-1 transcription activator protein Tat is phosphorylated in vitro by CDK2 and DNA-PK on Ser-16 residue and by PKR on Tat Ser-46 residue. Here we analyzed Tat phosphorylation in cultured cells and its functionality. Results Mass spectrometry
Andrey Ivanov, Xionghao Lin, Tatiana Ammosova, Andrey V. Ilatovskiy, Namita Kumari, Hatajai Lassiter, Nowah Afangbedji, Xiaomei Niu, Michael G. Petukhov, Sergei Nekhai   +9 more
doaj   +1 more source

Concomitant Presence of Hb Agrinio and - -Med Deletion in a Greek Male Patient with Hemoglobinopathy H: More Severe Phenotype and Literature Review

Hematology Reports, 2023
Hemoglobin (Hb) Agrinio is a rare non-deletional a-globin mutation observed almost exclusively in Greek, Spanish or other Mediterranean families. The clinical manifestations of a carrier of a single Hb Agrinio mutation (single heterozygosity) depend on ...
Michael D. Diamantidis, Stefania Pitsava, Omar Zayed, Ioanna Argyrakouli, Konstantinos Karapiperis, Christos Chatzoulis, Evangelos Alexiou, Achilles Manafas, Evangelos Tsangalas, Konstantinos Karakoussis   +9 more
doaj   +1 more source