Academic literature on the topic 'Mono-Hbb'

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Journal articles on the topic "Mono-Hbb"

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Guerra, Amaliris, Ariel Rivera, Nolan Hamilton, et al. "Differential Effects of Monoferric Transferrin Forms on Erythropoiesis Are Mediated By TFR2." Blood 142, Supplement 1 (2023): 3846. http://dx.doi.org/10.1182/blood-2023-189107.

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Transferrin (TF) is a bilobed 80kD glycoprotein with N- and C-lobe iron binding sites. TF circulates as four forms: unbound to iron (apo-TF), monoferric iron bound to the N-lobe or C-lobe (mono-TF) or to both lobes (holo-TF). All TF forms interact with TF receptor-1 (TFR1), which is ubiquitously expressed and serves as the main mechanism for cellular iron delivery. TF also interacts with TFR2 which is mainly expressed by hepatocytes and erythroid precursors to modulate cellular signaling events regulating hepcidin expression and erythropoiesis. We previously generated N-lobe blocked ( Tf N-bl)
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Koniali, Lola, Christina Flouri, Markela I. Kostopoulou та ін. "Evaluation of Mono- and Bi-Functional GLOBE-Based Vectors for Therapy of β-Thalassemia by HBBAS3 Gene Addition and Mutation-Specific RNA Interference". Cells 12, № 24 (2023): 2848. http://dx.doi.org/10.3390/cells12242848.

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Therapy via the gene addition of the anti-sickling βAS3-globin transgene is potentially curative for all β-hemoglobinopathies and therefore of particular clinical and commercial interest. This study investigates GLOBE-based lentiviral vectors (LVs) for βAS3-globin addition and evaluates strategies for an increased β-like globin expression without vector dose escalation. First, we report the development of a GLOBE-derived LV, GLV2-βAS3, which, compared to its parental vector, adds anti-sickling action and a transcription-enhancing 848-bp transcription terminator element, retains high vector tit
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Radtke, Stefan, Emily Fields, Gregory A. Newby, et al. "Efficient and Stable Makassar Base Editing in Nonhuman Primates." Blood 142, Supplement 1 (2023): 484. http://dx.doi.org/10.1182/blood-2023-178732.

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Sickle cell disease (SCD) is caused by a single nucleotide change in the β-globin chain. This nucleotide can be modified using adenine base editors (ABEs) to convert the defective SCD β-globin gene (HBB S) into the non-pathogenic Makassar β-globin (HBB M) variant. Proof-of-concept for this editing strategy in human and mouse hematopoietic stem cells (HSCs) has been previously demonstrated by transplantation after ex vivo modification as well as by in vivo adenoviral editing followed by selection. Here, we evaluated the feasibility, long-term efficiency, and safety of autologous ex vivo Makassa
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Chu, S. Haihua, Daisy Lam, Michael S. Packer, et al. "Adenine Base Editing of the Sickle Allele in CD34+ Hematopoietic Stem and Progenitor Cells Eliminates Hemoglobin S." Blood 136, Supplement 1 (2020): 47. http://dx.doi.org/10.1182/blood-2020-141805.

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While there are several small molecule, gene therapy, and gene editing approaches for treating sickle cell disease (SCD), these strategies do not result in the direct elimination of the causative sickle β-globin (HbS) variant itself. The reduction or complete removal of this pathologic globin variant and expression of normal β-hemoglobin (HbB) or other non-polymerizing β-globin variant may increase the likelihood of beneficial outcomes for SCD patients. Adenine base editors (ABEs) can precisely convert the mutant A-T base pair responsible for SCD to a G-C base pair, thus generating the hemoglo
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Malik, Jeffrey, Michael Getman, and Laurie A. Steiner. "The Histone Methyltransferase Setd8 Represses Gata2 Expression and Regulates Erythroid Maturation." Blood 124, no. 21 (2014): 4343. http://dx.doi.org/10.1182/blood.v124.21.4343.4343.

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Abstract The maturation of a committed erythroid progenitor to a functional red blood cell is a complex process that involves significant changes in gene expression during a time of rapid cell division and nuclear condensation. Posttranslational modifications of histone proteins are critical determinates of erythroid gene expression, however the histone modifications required to execute the erythroid-specific transcriptional program during maturation are incompletely understood. Setd8 is the sole histone methyltransferase in mammals capable of generating mono-methylated histone H4 lysine 20 (H
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Rivers, Angela, Maria Armila Ruiz, Kestis Vaitkus, Tatiana Kouznetsova, Joseph DeSimone та Donald Lavelle. "RN-1, a New and Potent Lsd-1 Inhibitor, Increases γ-Globin Expression In a Humanized Sickle Cell Mouse Model". Blood 122, № 21 (2013): 561. http://dx.doi.org/10.1182/blood.v122.21.561.561.

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Abstract Increased levels of fetal hemoglobin (HbF) decrease symptoms and increase life expectancy in Sickle Cell Disease (SCD). DNA Methyltransferase 1 (DNMT1) and Lysine Specific Demethylase-1, (LSD-1), a mono- and dimethyl-histone H3 K4 demethylating enzyme, are components of the DRED multiprotein complex, a repressor of γ-globin expression (Cui et al Mol Cell Biol 31:3298, 2011). While DNMT1 inhibitors are well known to induce high levels of HbF in non-human primates and SCD patients, it was recently shown that the LSD1 inhibitor tranylcypromine (TC) increased γ-globin expression in cultur
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Dissertations / Theses on the topic "Mono-Hbb"

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Kumar, Deepak. "Search for dark matter produced in association with a Higgs boson decaying to a pair of bottom quarks in proton-proton collisions at √s= 13 TeV using CMS 2017 and 2018 data." Thesis, 2023. https://etd.iisc.ac.in/handle/2005/6138.

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The discovery of the Higgs boson has opened a new portal to search for Dark Matter(DM). Since DM does not interact in the CMS detector, searches rely on the missing transverse energy in association with a visible SM particle. The final state of the Higgs boson+missing transverse energy is known as ”mono-Higgs”. In this thesis, mono-Higgs analysis has been performed in two categories; Boosted and Resolved. In the first part of the thesis, we present a search for DM when it is produced in association with a highly boosted Higgs boson decaying to a pair of bottom quarks using data collecte
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