Results 111 to 120 of about 358,486 (301)
MiR‐513a promotes human erythroid differentiation by modulating c‐Jun
FEBS Open Bio, EarlyView.During early human erythropoiesis, miR‐513a promoted erythroid differentiation in primary human CD34+ hematopoietic stem‐progenitor cells and human TF‐1 erythroleukemic cells by indirectly decreasing c‐Jun and phospho‐c‐Jun expression, which are associated with increased GATA1 expression.
MinJung Kim +11 more
wiley +1 more source
CRISPR's unwanted anniversary [PDF]
Science, 2019 There are key moments in the history of every disruptive technology that can make or break its public perception and acceptance. For CRISPR-based genome editing, such a moment occurred 1 year ago—an unsettling push into an era that will test how society decides to use this revolutionary technology.
openaire +2 more sources
Mass spectrometry based identification of AMP‐O‐Tris generated by Thermococcus onnurineus Cas10
FEBS Open Bio, EarlyView.Isolated Thermococcus onnurineus Cas10 generates the noncanonical ATP‐derived product AMP‐O‐Tris while in Tris‐containing buffer as identified via mass spectrometry, revealing relaxed nucleophile selectivity under isolated conditions. These findings suggest that multiprotein Csm complex assembly restricts Cas10 reactivity toward canonical cyclic ...
Su‐Jin Lee +6 more
wiley +1 more source
Exploratory Analysis of ELP1 Expression in Whole Blood From Patients With Familial Dysautonomia
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Background Familial dysautonomia (FD) is a hereditary neurodevelopmental disorder caused by aberrant splicing of the ELP1 gene, leading to a tissue‐specific reduction in ELP1 protein expression. Preclinical models indicate that increasing ELP1 levels can mitigate disease manifestations.
Alejandra González‐Duarte +13 more
wiley +1 more source
Developmental and Epileptic Encephalopathy due to Biallelic Pathogenic Variants in PIGM
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective PIGM encodes a critical enzyme in the glycosylphosphatidylinositol (GPI)‐anchor biosynthesis pathway. While promoter‐region mutations in PIGM have been associated with a relatively mild phenotype characterized by portal vein thrombosis and absence seizures, recent evidence suggests that coding‐region mutations result in a more severe
Júlia Sala‐Coromina +11 more
wiley +1 more source
Delivering CRISPR: a review of the challenges and approaches
Drug Delivery, 2018 Gene therapy has long held promise to correct a variety of human diseases and defects. Discovery of the Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR), the mechanism of the CRISPR-based prokaryotic adaptive immune system (CRISPR ...
Christopher A. Lino +3 more
doaj +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
Cardiac‐Derived ECM Microspheres for Enhanced hiPSC‐CMs Maturation
Advanced Functional Materials, EarlyView.Cardiac extracellular matrix microspheres derived from decellularized porcine heart provide a biomimetic 3D microenvironment for human induced pluripotent stem cell–derived cardiomyocytes (hiPSC‐CMs). This platform supports short‐ and long‐term culture, enhances structural organization, and promotes electrophysiological and functional maturation of ...
Jiazhu Xu +9 more
wiley +1 more source
Advanced Functional Materials, EarlyView.This study introduces VIVID (Vesicle In Vivo Identification using DNA), a qPCR‐based platform that tracks PCR‐amplifiable DNA tags loaded in the EVs for accurate and quantifiable EV biodistribution in vivo. ABSTRACT Extracellular vesicles (EVs) represent promising carriers for nucleic acid therapeutics, offering advantages over synthetic nanoparticles ...
Oscar Boyadjian +5 more
wiley +1 more source
Advanced Functional Materials, EarlyView.Butterfly wing scales are intricate cuticular functional nanosctructures. This perspective suggests that spatially varying material properties, cytoskeletal constraints, and growth‐driven mechanical instabilities shape the resulting nanoscale architectures created from single cells.
Anupama Prakash +10 more
wiley +1 more source