Results 21 to 30 of about 819,369 (331)

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

Science Advances, 2020
Editing the fetal γ-globin promoters in hematopoietic stem cells from sickle cell disease patients induces therapeutic γ-globin levels. Sickle cell disease (SCD) is caused by a single amino acid change in the adult hemoglobin (Hb) β chain that causes Hb ...
Leslie Weber, G. Frati, T. Felix, G. Hardouin, Antonio Casini, Clara Wollenschlaeger, Vasco Meneghini, C. Masson, A. de Cian, Anne Chalumeau, F. Mavilio, Mario Amendola, I. André-Schmutz, A. Cereseto, W. El Nemer, J. Concordet, C. Giovannangeli, M. Cavazzana, A. Miccio   +18 more
semanticscholar   +1 more source

Induction of fetal hemoglobin: Lentiviral shRNA knockdown of HBS1L in β0-thalassemia/HbE erythroid cells.

PLoS ONE, 2023
Imbalanced globin chain output contributes to thalassemia pathophysiology. Hence, induction of fetal hemoglobin in β-thalassemia and other β-hemoglobinopathies are of continuing interest for therapeutic approaches.
Sukanya Chumchuen, Orapan Sripichai, Natee Jearawiriyapaisarn, Suthat Fucharoen, Chayanon Peerapittayamongkol   +4 more
doaj   +1 more source

ZNF410 uniquely activates the NuRD component CHD4 to silence fetal hemoglobin expression

bioRxiv, 2020
Metazoan transcription factors typically regulate large numbers of genes. Here we identify via a CRISPR-Cas9 genetic screen ZNF410, a pentadactyl DNA binding protein that in human erythroid cells directly and measurably activates only one gene, the NuRD ...
Xianjiang Lan, Ren Ren, Ruopeng Feng, Lana C. Ly, Y. Lan, Zhe Zhang, Nicholas G. Aboreden, Kunhua Qin, J. Horton, Jeremy D. Grevet, Thiyagaraj Mayuranathan, O. Abdulmalik, C. Keller, B. Giardine, R. Hardison, M. Crossley, M. Weiss, Xiaodong Cheng, Junwei Shi, G. Blobel   +19 more
semanticscholar   +1 more source

Effect of hydroxyurea on G gamma chain fetal hemoglobin synthesis by sickle-cell disease patients

Brazilian Journal of Medical and Biological Research, 2003
Hydroxyurea is used for sickle-cell disease patients in order to increase fetal hemoglobin synthesis and consequently decrease the severity of pain episodes.
S.M. Teixeira, L.C. Cortellazzi, H.Z.W. Grotto   +2 more
doaj   +1 more source

Fetal Hemoglobin in Sickle Hemoglobinopathies: High HbF Genotypes and Phenotypes

Journal of Clinical Medicine, 2020
Fetal hemoglobin (HbF) usually consists of 4 to 10% of total hemoglobin in adults of African descent with sickle cell anemia. Rarely, their HbF levels reach more than 30%.
M. Steinberg
semanticscholar   +1 more source

The LSD1 inhibitor RN-1 recapitulates the fetal pattern of hemoglobin synthesis in baboons (P. anubis)

Haematologica, 2016
Increased fetal hemoglobin levels lessen the severity of symptoms and increase the lifespan of patients with sickle cell disease. Hydroxyurea, the only drug currently approved for the treatment of sickle cell disease, is not effective in a large ...
Angela Rivers, Kestis Vaitkus, Vinzon Ibanez, Maria Armila Ruiz, Ramasamy Jagadeeswaran, Yogen Saunthararajah, Shuaiying Cui, James D. Engel, Joseph DeSimone, Donald Lavelle   +9 more
doaj   +1 more source

HRI-regulated transcription factor ATF4 activates BCL11A transcription to silence fetal hemoglobin expression.

Blood, 2020
Reactivation of fetal hemoglobin remains a critical goal in the treatment of patients with sickle cell disease and β-thalassemia. Previously, we discovered that silencing of the fetal γ-globin gene requires the erythroid-specific eIF2α kinase HRI ...
Peng Huang, Scott A. Peslak, Xianjiang Lan, Eugene Khandros, Jennifer A. Yano, Malini Sharma, C. Keller, B. Giardine, Kunhua Qin, O. Abdulmalik, R. Hardison, Junwei Shi, G. Blobel   +12 more
semanticscholar   +1 more source

Hydroxyurea ameliorates mouse hypoxia through inducing embryonic hemoglobin

陆军军医大学学报, 2023
Objective To investigate the ameliorative effect and mechanism of hydroxyurea (HU) on hypoxia mice in a closed environment. Methods Sixty male C57BL/6J mice (6~8 weeks old, weighing 18~22 g) were randomly divided into normoxia control group (NC, n=10 ...
ZHOU Xiaoying, JIAO Li, CUI Yu, CUI Yu, LI Xiaoxu, YANG Yidong   +4 more
doaj   +1 more source

In vitro and in vivo induction of fetal hemoglobin with a reversible and selective DNMT1 inhibitor

Haematologica, 2020
Pharmacological induction of fetal hemoglobin (HbF) expression is an effective therapeutic strategy for the management of β-hemoglobinopathies such as sickle cell disease.
A. Gilmartin, Arthur Groy, Elizabeth R. Gore, C. Atkins, E. R. Long, M. Montoute, Zining Wu, W. Halsey, D. Mcnulty, D. Ennulat, Lourdes Rueda, M. Pappalardi, R. Kruger, M. McCabe, A. Raoof, R. Butlin, A. Stowell, Mark Cockerill, I. Waddell, D. Ogilvie, J. Luengo, A. Jordan, Andrew B. Benowitz   +22 more
semanticscholar   +1 more source

Breaking Free from Your Fetal Chains: A Case-Based Review of the Literature on Gamma Chain Variant Hemoglobinopathies [PDF]

Neonatal Medicine, 2023
Methemoglobinemia is a dyshemoglobinemia characterized by cyanosis and reduced oxygen saturation with increased methemoglobin values. The etiology may be congenital or acquired, with the latter being more common.
Pearl Mary Varughese, Sajan Thomas, Sheela Nampoothiri, Perraju Bendapudi   +3 more
doaj   +1 more source