Results 31 to 40 of about 151,779 (318)
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 ...
Rachael Davis +4 more
openaire +3 more sources
Identification and characterization of novel factors that act in the nonsense-mediated mRNA decay pathway in nematodes, flies and mammals [PDF]
, 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
BMC Biology, 2008 Background Vertebrate alpha (α)- and beta (β)-globin gene families exemplify the way in which genomes evolve to produce functional complexity. From tandem duplication of a single globin locus, the α- and β-globin clusters expanded, and then were ...
Warren Wesley C +7 more
doaj +1 more source
A dual reporter mouse model of the human β-globin locus: applications and limitations. [PDF]
PLoS ONE, 2012 The human β-globin locus contains the β-like globin genes (i.e. fetal γ-globin and adult β-globin), which heterotetramerize with α-globin subunits to form fetal or adult hemoglobin.
Petros Papadopoulos +8 more
doaj +1 more source
Mithramycin encapsulated in polymeric micelles by microfluidic technology as novel therapeutic protocol for beta-thalassemia [PDF]
, 2012 This report shows that the DNA-binding drug, mithramycin, can be efficiently encapsulated in polymeric micelles (PM-MTH), based on Pluronic® block copolymers, by a new microfluidic approach.
Brognara, Eleonora +8 more
core +2 more sources
The role of WDR5 in silencing human fetal globin gene expression
Haematologica, 2012 Background Histone H3 lysine 4 (K4) methylation has been linked with transcriptional activity in mammalian cells. The WD40-repeat protein, WDR5, is an essential component of the MLL complex that induces histone H3 K4 methylation, but the role of WDR5 in ...
Zhen Xu +9 more
doaj +1 more source
Reactivation of a developmentally silenced embryonic globin gene
Nature Communications, 2021 Globin loci harbor genes that are expressed embryonically and silenced postnatally. Here the authors show that zeta-globin silencing depends upon selective hypoacetylation of its TAD subdomain, which blocks its interaction with the alpha-globin super ...
Andrew J. King +25 more
doaj +1 more source
Cryptic transcripts from a ubiquitous plasmid origin of replication confound tests for cis-regulatory function. [PDF]
, 2012 A vast amount of research on the regulation of gene expression has relied on plasmid reporter assays. In this study, we show that plasmids widely used for this purpose constitutively produce substantial amounts of RNA from a TATA-containing cryptic ...
Hiraoka, Kei +3 more
core +2 more sources
Cell Reports, 2018 Summary: Fetal globin genes are transcriptionally silenced during embryogenesis through hemoglobin switching. Strategies to derepress fetal globin expression in the adult could alleviate symptoms in sickle cell disease and β-thalassemia.
Bjorg Gudmundsdottir +16 more
doaj +1 more source
The synthesis of hemoglobin in a cell-free system [PDF]
, 1958 The microsomal particles (1) have been implicated as the major sites of protein synthesis within the cells of a variety of tissues. The studies of Borsook, Zamecnik, Hultin, and others, with intact animals and various types of whole cell systems (see ...
Allen, Esther +2 more
core +2 more sources