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Artificial RNA Editing with ADAR for Gene Therapy

Current Gene Therapy, 2020
Editing mutated genes is a potential way for the treatment of genetic diseases. G-to-A mutations are common in mammals and can be treated by adenosine-to-inosine (A-to-I) editing, a type of substitutional RNA editing. The molecular mechanism of A-to-I editing involves the hydrolytic deamination of adenosine to an inosine base; this reaction is mediated
Sonali Bhakta, Toshifumi Tsukahara
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ADAR Gene Family and A-to-I RNA Editing: Diverse Roles in Posttranscriptional Gene Regulation

Progress in Molecular Biology and Translational Science, 2005
Publisher Summary The biological process of A-to-I RNA editing mediated by ADAR is discussed with new directions on potentially novel targets, including the widely expressed Alu retrotransposable elements found in noncoding regions of mRNA. Many events take place after the de novo synthesis of an RNA transcript, leading to alterations from its gene ...
Louis, Valente, Kazuko, Nishikura
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Involvement of PU.1 in mouse adar-1 gene transcription induced by high-dose esiRNA

International Journal of Biological Macromolecules, 2009
Adar-1 gene plays an important role in the negative regulation of RNA interference. We previously showed that increased adar-1 mRNA level was associated with the rebound of gene expression after RNAi suppression. In this study, we identified a PU.1 binding site upstream from transcription start point of adar-1 gene and is essential for the promoter ...
Tongde, Wu   +4 more
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Variable clinical phenotype in two siblings with Aicardi-Goutières syndrome type 6 and a novel mutation in the ADAR gene

European Journal of Paediatric Neurology, 2018
Aicardi-Goutières syndrome (AGS) is a hereditary inflammatory encephalopathy resulting in severe neurological damage in the majority of cases. We report on two siblings with AGS6 due to compound heterozygosity for a known and a novel mutation in the ADAR gene and a strikingly variable phenotype.
Lisa, Schmelzer   +9 more
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[Analysis of ADAR gene variant in a Chinese pedigree affected with dyschromatosis symmetrica hereditaria].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics, 2022
To analyze the clinical features and genetic basis for a Chinese pedigree affected with hereditary dyschromatosis symmetrica hereditaria (DSH).Peripheral blood samples of the proband and his mother were collected and subjected to PCR and Sanger sequencing.The patient has conformed to the typical pattern of DSH and manifested with hyperpigmentation ...
Changyin, Wang   +6 more
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A frameshift mutation in the ADAR gene in a Korean family with dyschromatosis symmetrica hereditaria

European Journal of Dermatology, 2014
Dyschromatosis symmetrica hereditaria (DSH; MIM 127400) is a rare autosomal dominant skin disorder characterized by a mixture of hyper- and hypopigmented macules on the dorsal surface of the extremities and face. Onset of the disease is usually during infancy or early childhood. Dyschromatosis stops spreading before adolescence and lasts for life.
Young Bok Lee   +6 more
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Insights into ADAR gene complement, expression patterns, and RNA editing landscape in Chlamys farreri

Fish & Shellfish Immunology
Adenosine Deaminases Acting on RNA (ADARs) are evolutionarily conserved enzymes known to convert adenosine to inosine in double-stranded RNAs and participate in host-virus interactions. Conducting a meta-analysis of available transcriptome data, we identified and characterised eight ADAR transcripts in Chlamys farreri, a farmed marine scallop ...
Enrico Bortoletto   +5 more
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