Results 71 to 80 of about 204,351 (287)
Nucleic Acids Research, 2020 Eukaryotic protein synthesis generally initiates at a start codon defined by an AUG and its surrounding Kozak sequence context, but the quantitative importance of this context in different species is unclear.
Edward W. J. Wallace +9 more
semanticscholar +1 more source
Advanced Functional Materials, EarlyView.Lipid nanoparticles (LNPs) are optimized to co‐deliver Cas9‐encoding messenger RNA (mRNA), a single guide RNA (sgRNA) targeting the endogenous cystic fibrosis transmembrane conductance regulator (CFTR) gene, and homologous linear double‐stranded donor DNA (ldsDNA) templates encoding CFTR.
Ruth A. Foley +12 more
wiley +1 more source
Functional rescue of dystrophin deficiency in mice caused by frameshift mutations using Campylobacter jejuni Cas9 [PDF]
, 2018 Duchenne muscular dystrophy (DMD) is a fatal, X-linked muscle wasting disease caused by mutations in the DMD gene. In 51% of DMD cases, a reading frame is disrupted because of deletion of several exons.
Cappellari, O +9 more
core +2 more sources
An Expanded Toolbox for Versatile Chemical Editing of Adeno‐Associated Virus
Angewandte Chemie, EarlyView.We describe technology to introduce diverse non‐natural chemical functionalities site‐specifically into the capsid of adeno‐associated virus through genetic code expansion, and using them to engineer this leading vector for human gene therapy for enhanced tissue specificity and reduced immunogenicity Abstract Site‐specific incorporation of noncanonical
Quan Pham +6 more
wiley +2 more sources
MicroRNA-mediated repression of nonsense mRNAs
eLife, 2014 Numerous studies have established important roles for microRNAs (miRNAs) in regulating gene expression. Here, we report that miRNAs also serve as a surveillance system to repress the expression of nonsense mRNAs that may produce harmful truncated ...
Ya Zhao +5 more
doaj +1 more source
Advanced Science, EarlyView.Phosphorylation of Optineurin by TBK1 induces the formation of filaments that condensate upon binding to linear polyubiquitin. Membrane‐anchored LC3 partitions into these condensates, suggesting that phase separation of filamentous Optineurin with ubiquitylated cargo promotes the sequestration of cargo and its subsequent alignment with LC3‐positive ...
Maria G. Herrera +10 more
wiley +1 more source
Aberrant Splicing Signatures Underpin Oligodendrocyte Damage in ALS and Neuron Loss in FTD
Advanced Science, EarlyView.Integrating bulk and single‐nucleus RNA‐seq from ALS and FTD patients, we revealed distinct cellular dysfunctions, with severe oligodendrocyte damage in ALS and pronounced neuron loss in FTD. We identified cell‐type‐specific aberrant splicing events as potential biomarkers with robust classification performance, some of which produce de novo peptides ...
Chen Du +6 more
wiley +1 more source
, 2014 The degradation of human ether-a-go-go-related gene (hERG, KCNH2) transcripts containing premature termination codon (PTC)mutations by nonsense-mediatedmRNA decay (NMD) is an importantmechanismof long QT syndrome type 2 (LQT2).
Gong, Qiuming +2 more
core +1 more source
Molecular Therapy: Methods & Clinical Development, 2020 Nonsense-mediated decay (NMD) is a major pathogenic mechanism underlying a diversity of genetic disorders. Nonsense variants tend to lead to more severe disease phenotypes and are often difficult targets for small molecule therapeutic development as a ...
Steven Erwood +6 more
doaj +1 more source
Precursor RNAs Harboring Nonsense Codons Accumulate Near the Site of Transcription [PDF]
Molecular Cell, 2001 Messenger RNAs containing premature termination codons (PTCs) are selectively eliminated by nonsense-mediated mRNA decay (NMD). Paradoxically, although cytoplasmic ribosomes are the only known species capable of PTC recognition, in mammals many PTC-containing mRNAs are apparently eliminated prior to release from the nucleus.
Mühlemann, Oliver +7 more
openaire +2 more sources