Results 21 to 30 of about 1,339,121 (337)

Elevating fetal hemoglobin - recently discovered regulators and mechanisms.

Blood
It has been known for over half a century that humans produce different forms of hemoglobin, a tetramer of α- and β-like hemoglobin chains, throughout ontogeny. The switch from fetal to adult hemoglobin occurs around the time of birth when erythropoiesis
G. Blobel, Eugene Khandros
semanticscholar   +2 more sources

Potent and uniform fetal hemoglobin induction via base editing

Nature Genetics, 2023
A comparison of fetal hemoglobin gene editing strategies using human sickle cell disease donor cells and in vivo transplantation finds that adenine base editing of the –175A>G site in the γ-globin gene promoters results in durable and potent expression ...
Thiyagaraj Mayuranathan, Gregory A. Newby, Ruopeng Feng, Yu Yao, Kalin Mayberry, C. Lazzarotto, Yichao Li, Rachel M. Levine, Nikitha Nimmagadda, Erin Dempsey, G. Kang, Shaina N. Porter, Phillip A. Doerfler, Jingjing Zhang, YoonJeong Jang, Jingjing Chen, Henry W. Bell, M. Crossley, S. Bhoopalan, Akshay Sharma, J. Tisdale, Shondra M. Pruett-Miller, Yong Cheng, S. Tsai, David R. Liu, M. Weiss, Jonathan S. Yen   +26 more
semanticscholar   +1 more source

Fetal Hemoglobin Regulation in Beta-Thalassemia.

Hematology/Oncology Clinics of North America, 2023
β-thalassemia is caused by mutations that reduce β-globin production, causing globin chain imbalance, ineffective erythropoiesis, and consequent anemia. Increased fetal hemoglobin (HbF) levels can ameliorate the severity of β-thalassemia by compensating ...
Henry Y. Lu, S. Orkin, V. Sankaran
semanticscholar   +1 more source

Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression

Nature Communications, 2022
Sickle cell disease and β-thalassemia affect the production of the adult β-hemoglobin chain. The clinical severity is lessened by mutations that cause fetal γ-globin expression in adult life (i.e., the hereditary persistence of fetal hemoglobin ...
P. Antoniou, G. Hardouin, Pierre Martinucci, G. Frati, T. Felix, A. Chalumeau, Letizia Fontana, J. Martin, C. Masson, M. Brusson, Giulia Maule, M. Rosello, C. Giovannangeli, V. Abramowski, J. de Villartay, J. Concordet, F. Del Bene, W. El Nemer, Mario Amendola, M. Cavazzana, A. Cereseto, Oriana Romano, A. Miccio   +22 more
semanticscholar   +1 more source

Pharmacological Induction of Fetal Hemoglobin in β-Thalassemia and Sickle Cell Disease: An Updated Perspective

Pharmaceuticals, 2022
A significant amount of attention has recently been devoted to the mechanisms involved in hemoglobin (Hb) switching, as it has previously been established that the induction of fetal hemoglobin (HbF) production in significant amounts can reduce the ...
R. Bou-Fakhredin, L. De Franceschi, I. Motta, M. Cappellini, A. Taher   +4 more
semanticscholar   +1 more source

The Clinical Approach toward Hereditary Persistence of Fetal Hemoglobin: A Case Report

Iranian Journal of Allergy, Asthma and Immunology, 2022
Fetal hemoglobin is the principal hemoglobin in the human fetus, and the adult levels of fetal hemoglobin (HbF) are less than 1% of total hemoglobin.
Afshin Ghaderi, Tahereh Bakhtiari, Saeid Jokar, Abbas Eshraghi   +3 more
doaj   +1 more source

A Cell-Permeant Nanobody-Based Degrader That Induces Fetal Hemoglobin

bioRxiv, 2022
Proximity-based strategies to degrade proteins have enormous therapeutic potential in medicine, but the technologies are limited to proteins for which small molecule ligands exist.
Fangfang Shen, Ge Zheng, Mekedlawit T Setegne, Karin Tenglin, Manizheh Izadi, Henry Xie, Liting Zhai, S. Orkin, Laura M. K. Dassama   +8 more
semanticscholar   +1 more source

Single-nucleotide–level mapping of DNA regulatory elements that control fetal hemoglobin expression

Nature Genetics, 2021
Pinpointing functional noncoding DNA sequences and defining their contributions to health-related traits is a major challenge for modern genetics. We developed a high-throughput framework to map noncoding DNA functions with single-nucleotide resolution ...
Li Cheng, Yichao Li, Qian Qi, Peng Xu, Ruopeng Feng, L. Palmer, Jingjing Chen, Ruiqiong Wu, Tiffany Yee, Jingjing Zhang, Yu Yao, Akshay Sharma, R. Hardison, M. Weiss, Yong Cheng   +14 more
semanticscholar   +1 more source

Activation of γ-globin gene expression by GATA1 and NF-Y in hereditary persistence of fetal hemoglobin

Nature Genetics, 2021
Hereditary persistence of fetal hemoglobin (HPFH) ameliorates β-hemoglobinopathies by inhibiting the developmental switch from γ-globin (HBG1/HBG2) to β-globin (HBB) gene expression.
Phillip A. Doerfler, Ruopeng Feng, Yichao Li, L. Palmer, Shaina N. Porter, Henry W. Bell, M. Crossley, Shondra M. Pruett-Miller, Yong Cheng, M. Weiss   +9 more
semanticscholar   +1 more source

Multiplex CRISPR/Cas9 genome editing in hematopoietic stem cells for fetal hemoglobin reinduction generates chromosomal translocations

Molecular Therapy: Methods & Clinical Development, 2021
Sickle cell disease and β-thalassemia are common monogenic disorders that cause significant morbidity and mortality globally. The only curative treatment currently is allogeneic hematopoietic stem cell transplantation, which is unavailable to many ...
Clare Samuelson, S. Radtke, Haiying Zhu, Mallory J Llewellyn, Emily Fields, Savannah M. Cook, Meei-Li W. Huang, K. Jerome, H. Kiem, Olivier Humbert   +9 more
semanticscholar   +1 more source