Results 201 to 210 of about 287,741 (287)

Reducing off-target expression of mRNA therapeutics and vaccines in the liver with microRNA binding sites. [PDF]

Mol Ther Methods Clin Dev
Parrett BJ, Yamaoka S, Barry MA.
europepmc   +1 more source

Unraveling the Molecular Mechanisms of Glioma Recurrence: A Study Integrating Single‐Cell and Spatial Transcriptomics

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Glioma recurrence severely impacts patient prognosis, with current treatments showing limited efficacy. Traditional methods struggle to analyze recurrence mechanisms due to challenges in assessing tumor heterogeneity, spatial dynamics, and gene networks.
Lei Qiu, Yinjiao Fei, Jiaxuan Ding, Kexin Shi, Jinyan Luo, Yuchen Zhu, Xingjian Sun, Gefei Jiang, Yuandong Cao, Weilin Xu, Shu Zhou   +10 more
wiley   +1 more source

Lipid Nanoparticles With Fine-Tuned Composition Show Enhanced Colon Targeting as a Platform for mRNA Therapeutics. [PDF]

Adv Sci (Weinh)
Rampado R, Naidu GS, Karpov O, Goldsmith M, Sharma P, Ezra A, Stotsky L, Breier D, Peer D.   +8 more
europepmc   +1 more source

TBK1‐Associated Primary Lateral Sclerosis Followed by Right Temporal Variant Frontotemporal Dementia

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT We report a 58‐year‐old woman with a novel splice‐site variant in the TANK‐binding kinase 1 (TBK1:c.993–2A>C p.Ala332TyrfsTer39) who sequentially developed primary lateral sclerosis (PLS) followed by right temporal variant frontotemporal dementia (rtvFTD). Neuroimaging demonstrated right anterior temporal atrophy before cognitive symptoms, and
Tomoyasu Matsubara, Naoki Kihara, Satoko Miyatake, Koji Fujita, Konoka Tachibana, Ryosuke Miyamoto, Hiroki Yamazaki, Yusuke Osaki, Nazere Keyoumu, Yuki Kuwano, Nobutoshi Morimoto, Suzuran Saito, Eriko Koshimizu, Yoichi Otomi, Kenji Ishibashi, Masafumi Harada, Naomichi Matsumoto, Hiroyuki Morino, Yuishin Izumi   +18 more
wiley   +1 more source

"Goldilocks Modifications" for mRNA Therapeutics Won the Nobel Prize. [PDF]

Nucleic Acid Ther
Li L.
europepmc   +1 more source

Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site‐Specific Integration of CFTR and Mutation‐Agnostic Disease Rescue

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, Paul G. Ayoub, Vrishti Sinha, Colin Juett, Alicia Sanoyca, Emily C. Duggan, Lindsay E. Lathrop, Priyanka Bhatt, Kevin Coote, Beate Illek, Brigitte N. Gomperts, Donald B. Kohn, Steven J. Jonas   +12 more
wiley   +1 more source

Analytical Ultracentrifugation to Assess the Quality of LNP-mRNA Therapeutics. [PDF]

Int J Mol Sci
Guerrini G, Mehn D, Scaccabarozzi D, Gioria S, Calzolai L.   +4 more
europepmc   +1 more source

Microporous Microgel Assemblies Facilitating the Recruitment and Osteogenic Differentiation of Progenitor Cells for Bone Regeneration

Advanced Functional Materials, EarlyView.
There is a significant need for biomaterials with well‐defined stability and bioactivity to support tissue regeneration. In this study, we developed a tunable microgel platform that enables the decoupling of stiffness from porosity, thereby promoting bone regeneration.
Silvia Pravato, Haishuo Guan, Bianca M. Carrara, Mira Jacobs, Matteo Monticelli, Lukas Moser, Thomas Moragues, Andrew J. deMello, Tomaso Zambelli, Martin Ehrbar   +9 more
wiley   +1 more source

Implementation of Drug‐Induced Rhabdomyolysis and Acute Kidney Injury in Microphysiological System

Advanced Functional Materials, EarlyView.
A modular Muscle–Kidney proximal tubule‐on‐a‐chip integrates 3D skeletal muscle and renal proximal tubule tissues to model drug‐induced rhabdomyolysis and acute kidney injury. The coculture system enables dynamic tissue interaction, functional contraction monitoring, and quantification of nephrotoxicity, revealing drug side effect‐induced metabolic ...
Jaesang Kim, Zhuo Feng Lee, Gi‐Dong Sim, Sejoong Kim, Jessie S. Jeon   +4 more
wiley   +1 more source

Development of Lipid Nanoparticle Formulation for the Repeated Administration of mRNA Therapeutics. [PDF]

Biomater Res
Lee Y, Jeong M, Lee G, Park J, Jung H, Im S, Lee H.   +6 more
europepmc   +1 more source