Results 161 to 170 of about 971,477 (354)
Epilepsy‐Associated Variants of a Single SCN1A Codon Exhibit Divergent Functional Properties
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Pathogenic variants in SCN1A, which encodes the voltage‐gated sodium channel NaV1.1, are associated with multiple epilepsy syndromes exhibiting a range of clinical severity. SCN1A variants are reported in different syndromes, including Dravet syndrome, which is associated with loss‐of‐function, whereas neonatal/infantile‐onset ...
Lanie N. Liebovitz +3 more
wiley +1 more source
Production of high-titer helper-free retroviruses by transient transfection.
Proceedings of the National Academy of Sciences of the United States of America, 1993 Warren S. Pear +3 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
Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA
Gene Therapy, 1999 Feng Liu, Y. Song, Dexi Liu
semanticscholar +1 more source
Unleashing the Power of Machine Learning in Nanomedicine Formulation Development
Advanced Functional Materials, EarlyView.A random forest machine learning model is able to make predictions on nanoparticle attributes of different nanomedicines (i.e. lipid nanoparticles, liposomes, or PLGA nanoparticles) based on microfluidic formulation parameters. Machine learning models are based on a database of nanoparticle formulations, and models are able to generate unique solutions
Thomas L. Moore +7 more
wiley +1 more source
Constitutively Active Galpha q and Galpha 13 Trigger Apoptosis through Different Pathways [PDF]
, 1997 We investigated the effect of expression of constitutively active Galpha mutants on cell survival. Transfection of constitutively active Galphaq and Galpha13 in two different cell lines caused condensation of genomic DNA and nuclear fragmentation ...
Althoefer, Henning +2 more
core
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
Adenovirus E1A Activation Domain Regulates H3 Acetylation Affecting Varied Steps in Transcription at Different Viral Promoters. [PDF]
, 2018 How histone acetylation promotes transcription is not clearly understood. Here, we confirm an interaction between p300 and the adenovirus 2 large E1A activation domain (AD) and map the interacting regions in E1A by observing colocalization at an ...
Berk, Arnold J +4 more
core
Advanced Functional Materials, EarlyView.xxxx. ABSTRACT Glioblastoma (GBM) remains one of the most lethal brain cancers, with median survival rarely exceeding 15 months after diagnosis. Interleukin‐12 (IL‐12) is a potent immunostimulatory cytokine capable of reshaping the tumor microenvironment (TME), yet its clinical translation is hindered by systemic toxicity and short half‐life. RNA‐based
Fatima Hameedat +11 more
wiley +1 more source
Development of Stable Cell Lines for the Production of Hematopoietic Stem Cell Targeted Exosomes [PDF]
, 2018 Exosomes represent a promising new approach to targeted drug delivery. Current research explores the ability of these naturally occurring nanoparticles to transport therapeutic cargo to specific tissues of the body and to subsequently enter the cells of ...
Beard, Anja, Ehlinger, Zach
core +1 more source