Results 91 to 100 of about 173,792 (325)

Targeting eRNA‐Producing Super‐Enhancers Regulates TNFα Expression and Mitigates Chronic Inflammation in Mice and Patient‐Derived Immune Cells

Advanced Science, EarlyView.
Targeting eRNAs from TNFα‐associated super‐enhancers with ASOs effectively reduces TNFα expression and inflammatory responses in both mouse models and patient‐derived immune cells. The conserved nature of these regulatory regions between species highlights the potential of eRNA‐targeted approaches as a therapeutic strategy for chronic inflammatory ...
Minjeong Cho, Su Min Kim, Jiyeon Lee, Oh Chan Kwon, Wonjin Woo, Eunji Lee, Hyo Jin Park, Yeongun Lee, So Hee Dho, Tae‐Kyung Kim, Min‐Chan Park, Richard A. Flavell, Lark Kyun Kim   +12 more
wiley   +1 more source

Inhibition of G9a methyltransferase stimulates fetal hemoglobin production by facilitating LCR/γ-globin looping.

Blood, 2015
Induction of fetal hemoglobin (HbF) production in adult erythrocytes can reduce the severity of sickle cell disease and β-thalassemia. Transcription of β-globin genes is regulated by the distant locus control region (LCR), which is brought into direct ...
I. Krivega, C. Byrnes, Jaira F. de Vasconcellos, Y. T. Lee, Megha Kaushal, A. Dean, Jeffery L. Miller   +6 more
semanticscholar   +1 more source

Engineering Globin Gene Expression [PDF]

Molecular Therapy - Methods & Clinical Development, 2019
Hemoglobinopathies, including sickle cell disease and thalassemia, are among the most common inherited genetic diseases worldwide. Due to the relative ease of isolating and genetically modifying hematopoietic stem and progenitor cells, recent gene editing and gene therapy strategies have progressed to clinical trials with promising outcomes; however ...
Jörg Bungert, Eliot M. Gunn, Aishwarya Gurumurthy, Mir A. Hossain, Rachael Davis   +4 more
openaire   +4 more sources

Divergent Delivery and Expression Kinetics of Lipid and Polymeric Nanoparticles across mRNA Modalities

Advanced Science, EarlyView.
Self‐amplifying (saRNA), linear (linRNA), and circular (circRNA) mRNAs are compared under standardized conditions using lipid nanoparticles (LNPs) and pABOL polymer. saRNA achieved superior expression, while linRNA and circRNA performance varied based on untranslated region elements and delivery method.
Irafasha C. Casmil, Josh J. Friesen, Nuthan V. Bathula, Anneke Strumpel, Chia Hao Ho, Ilana Guez, Kristen Y. S. Kong, Andrew J. Varley, Shigeki J. Miyake‐Stoner, Parinaz Aliahmad, Nathaniel S. Wang, Andrew J. Geall, Anna K. Blakney   +12 more
wiley   +1 more source

Quantitation of human gamma globin genes and gamma globin mRNA with purified gamma globin complementary DNA. [PDF]

Journal of Clinical Investigation, 1976
Complementary DNA (cDNA) specific for gamma-globin nucleotide sequences has been prepared by hybridizing total cDNA made from cord blood messenger RNA (mRNA) as template to an excess of normal adult human globin mRNA and recovering the single-stranded cDNA from hydroxylapatite.
Clayton Natta, Arthur Bank, Francesco Ramirez, Joyce V. O'Donnell   +3 more
openaire   +3 more sources

Peripheral Blood Gene Expression Profiling and Prognostic Significance for the Course of Interstitial Lung Disease in Patients With Systemic Sclerosis

Arthritis &Rheumatology, EarlyView.
Objective We used data from the placebo arm of the Safety and Efficacy of Nintedanib in Systemic Sclerosis (SENSCIS) trial to determine the prognostic/predictive significance of peripheral blood cell (PBC) transcript modules for the course of forced vital capacity (FVC) in patients with systemic sclerosis–associated interstitial lung disease (SSc‐ILD ...
Shervin Assassi, Christopher P. Denton, Matthias Zwick, Ramona Schmid, Carina Ittrich, Tobias Litzenburger, Sudha Visvanathan   +6 more
wiley   +1 more source

The diversity of globin‐coupled sensors [PDF]

FEBS Letters, 2003
The recently discovered globin‐coupled sensors (GCSs) are heme‐containing two‐domain transducers distinct from the PAS domain superfamily. We have identified an additional 22 GCSs with varying multi‐domain C‐terminal transmitters through a search of the complete and incomplete microbial genome datasets.
Maqsudul Alam, Shaobin Hou, Tracey Allen K. Freitas   +2 more
openaire   +3 more sources

CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells.

PLoS ONE, 2019
Sickle Cell Disease and ß-thalassemia, which are caused by defective or deficient adult ß-globin (HBB) respectively, are the most common serious genetic blood diseases in the world. Persistent expression of the fetal ß-like globin, also known as 𝛾-globin,
Jennifer E Chung, Wendy Magis, Jonathan Vu, Seok-Jin Heo, Kirmo Wartiovaara, Mark C Walters, Ryo Kurita, Yukio Nakamura, Dario Boffelli, David I K Martin, Jacob E Corn, Mark A DeWitt   +11 more
doaj   +1 more source

siRNA-mediated reduction of α-globin results in phenotypic improvements in β-thalassemic cells

Haematologica, 2008
β-thalassemia is an inherited hemoglobinopathy caused by defective synthesis of the β-globin chain of hemoglobin, leading to imbalanced globin chain synthesis.
Hsiao Phin Joanna Voon, Hady Wardan, Jim Vadolas   +2 more
doaj   +1 more source

Advanced Imaging and Cytometric Techniques to Characterize Lipid Accumulation in Wolman Disease

Cytometry Part A, EarlyView.
ABSTRACT Wolman disease (WD) is a severe lysosomal storage disorder characterized by fatal lipid accumulation caused by the deficiency of a lipid metabolic enzyme, Lysosomal Acid Lipase (LAL), involved in the lysosomal hydrolysis of cholesterols and triglycerides.
Marine Laurent, Jérémie Cosette, Giulia Pavani, Sarah Bayol, Christine Jenny, Rim Harb, Julie Oustelandt, Anais Brassier, Daniel Stockholm, Mario Amendola   +9 more
wiley   +1 more source