Results 51 to 60 of about 136,712 (243)

Enhanced Intracellular Stability and Translation Efficiency of mRNA Drugs by a 2‐arm mRNA Platform

open access: yesAdvanced Science, EarlyView.
We constructed a 2‐arm mRNA, characterized by a unique topology formed through the dimerization of two mRNA 3’ tails. The 2‐arm mRNA improves 3’ tail stability and resistance to nuclease degradation, resulting in an intracellular half‐life of up to 65 h. This method substantially enhances the translation capacity of mRNA drugs.
Xucong Teng   +5 more
wiley   +1 more source

Controlling α-globin: a review of α-globin expression and its impact on β-thalassemia

open access: yesHaematologica, 2008
Synthesis of α-globin and α-globin subunits of hemoglobin occurs at high levels during erythrocyte differentiation in a tightly controlled and coordinated fashion.
Hsiao Phin Joanna Voon, Jim Vadolas
doaj   +1 more source

βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid Cells

open access: yesMolecular Therapy: Methods & Clinical Development, 2020
Lentiviral addition of βT87Q-globin, a modified β-globin with an anti-sickling mutation, is currently being used in gene therapy trials for sickle cell disease (SCD) and β-thalassemia patients.
Selami Demirci   +10 more
doaj   +1 more source

A long and complex enhancer activates transcription of the gene coding for the highly abundant immediate early mRNA in murine cytomegalovirus [PDF]

open access: yes, 1985
Using the simian virus 40 "enhancer trap" approach, we have identified a transcription enhancer located just upstream of the major immediate early gene of murine cytomegalovirus. This enhancer has several striking properties.
Dorsch-Häsler, Karoline   +5 more
core   +1 more source

Genetics and Genomics in Sickle Cell Disease in Africa. [PDF]

open access: yesAm J Hematol
ABSTRACT Advanced genomic technologies are revolutionizing our ability to understand complex diseases. Large‐scale population studies are needed to realize the potential of using individual genetic information to personalize treatments for better patient outcomes for chronic non‐communicable diseases, such as sickle cell disease (SCD).
Nkya S, Makani J, Flanagan JM.
europepmc   +2 more sources

Identification and characterization of novel factors that act in the nonsense-mediated mRNA decay pathway in nematodes, flies and mammals [PDF]

open access: yes, 2014
Nonsense-mediated mRNA decay (NMD) is a surveillance mechanism that degrades mRNAs harboring premature termination codons (PTCs). We have conducted a genome-wide RNAi screen in Caenorhabditis elegans that resulted in the identification of five novel NMD ...
Ailion M   +8 more
core   +2 more sources

Combined Long‐Read Genome and Transcriptome Sequencing Establishes Novel Variants in MEGF8 as the Cause for Carpenter Syndrome Type 2

open access: yesAmerican Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Carpenter syndrome type 2 (CRPT2) is a rare autosomal recessive disease mainly characterized by craniosynostosis and polysyndactyly. CRPT2 is the rarer subtype of Carpenter syndrome (CRPTS) and is caused by biallelic variants in the multiple epidermal growth factor‐like domains 8 gene (MEGF8).
Kiana Rashidi   +11 more
wiley   +1 more source

Transcriptomic signatures reveal systemic adaptations and immune modulation in response to training and competitive racing in horses

open access: yesEquine Veterinary Journal, EarlyView.
Abstract Background The molecular mechanisms underlying adaptation to physical exertion and racing stress in horses remain incompletely understood. Peripheral blood transcriptomics offers a minimally invasive method to monitor systemic responses to exercise and identify biomarkers of adaptation or overload. Objectives To evaluate transcriptomic changes
Izabela Dąbrowska   +4 more
wiley   +1 more source

The Globin Gene Family in Arthropods: Evolution and Functional Diversity

open access: yesFrontiers in Genetics, 2020
Globins are small heme-proteins that reversibly bind oxygen. Their most prominent roles in vertebrates are the transport and storage of O2 for oxidative energy metabolism, but recent research has suggested alternative, non-respiratory globin functions ...
Andreas Prothmann   +8 more
doaj   +1 more source

Regulation of Gγ-globin gene by ATF2 and its associated proteins through the cAMP-response element. [PDF]

open access: yesPLoS ONE, 2013
The upstream Gγ-globin cAMP-response element (G-CRE) plays an important role in regulating Gγ-globin expression through binding of ATF2 and its DNA-binding partners defined in this study.
Li Liu   +6 more
doaj   +1 more source

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