Results 91 to 100 of about 451,143 (385)

SNUPN‐Related Muscular Dystrophy: Novel Phenotypic, Pathological and Functional Protein Insights

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective SNUPN‐related muscular dystrophy or LGMDR29 is a new entity that covers from a congenital or childhood onset pure muscular dystrophy to more complex phenotypes combining neurodevelopmental features, cataracts, or spinocerebellar ataxia. So far, 12 different variants have been described.
Nuria Muelas, Pablo Iruzubieta, Alberto Damborenea, Laura Pérez‐Fernández, Inmaculada Azorín, Juan Carlos Jiménez García, Ana Töpf, Pilar Martí, Lorena Fores‐Toribio, María Manterola, Rosana Blanco‐Mañez, Oihane Pikatza‐Menoio, Sonia Alonso‐Martín, Volker Straub, Aitziber L. Cortajarena, Adolfo López de Munain, David De Sancho, Lorea Blázquez, Juan J. Vilchez   +18 more
wiley   +1 more source

Reconfigurable Nucleic Acid Nanoparticles with Therapeutic RNAi Responses to Intracellular Disease Markers

Advanced Functional Materials, EarlyView.
Many known diseases arise from dysregulated gene expression, and differentially expressed genes can serve as biomarkers to distinguish diseased cells from healthy tissues. In this study, reconfigurable nucleic acid nanoparticles (recNANPs) are introduced that can detect overexpressed cancer biomarkers and subsequently release RNAi inducers to silence ...
Yelixza I. Avila, Anh Ha, Morgan R. Chandler, Nathalia Leal Santos, Taejin Kim, Hannah S. Newton, Marina A. Dobrovolskaia, Kirill A. Afonin   +7 more
wiley   +1 more source

Unanticipated differences between α- and γ-diaminobutyric acid-linked hairpin polyamide-alkylator conjugates [PDF]

, 2006
Hairpin polyamide–chlorambucil conjugates containing an {alpha}-diaminobutyric acid ({alpha}-DABA) turn moiety are compared to their constitutional isomers containing the well-characterized {gamma}-DABA turn.
Chou, C. James, Dervan, Peter B., Farkas, Michelle E., Gottesfeld, Joel M., Tsai, Sherry M.   +4 more
core   +4 more sources

FeDSNP‐Pa Nanoassemblies: A Triple‐Action Therapeutic Strategy Targeting Oxidative Stress, Inflammation, and Pyroptosis for Retinal Ganglion Cell Protection in Glaucoma

Advanced Functional Materials, EarlyView.
FeDSNP‐Pa, a metallized nanoparticle loaded with sodium pyruvate (Pa), exerts triple therapeutic effects by scavenging reactive oxygen species (ROS), suppressing inflammatory responses, and inhibiting pyroptosis signaling pathways. This multifunctional neuroprotective strategy protecting retinal ganglion cells (RGCs) from elevated intraocular pressure ...
Yukun Wu, Xue Li, Tong Wu, Dan Ji, Yugang Bai, Xuezhi Zhou   +5 more
wiley   +1 more source

Nucleic acid-induced NADase activation of a short Sir2-associated prokaryotic Argonaute system

Cell Reports
Summary: Inhibition of nucleic acid targets is mediated by Argonaute (Ago) proteins guided by RNA or DNA. Although the mechanisms underpinning the functions of eukaryotic and “long” prokaryotic Ago proteins (pAgos) are well understood, those for short ...
Dapeng Sun, Kaixiang Zhu, Linyue Wang, Zhixia Mu, Kang Wu, Lei Hua, Bo Qin, Xiaopan Gao, Yumei Wang, Sheng Cui   +9 more
doaj   +1 more source

Computational investigation of locked nucleic acid (LNA) nucleotides in the active sites of DNA polymerases by molecular docking simulations. [PDF]

PLoS ONE, 2014
Aptamers constitute a potential class of therapeutic molecules typically selected from a large pool of oligonucleotides against a specific target. With a scope of developing unique shorter aptamers with very high biostability and affinity, locked nucleic
Vasanthanathan Poongavanam, Praveen K Madala, Torben Højland, Rakesh N Veedu   +3 more
doaj   +1 more source

Structure motivator: a tool for exploring small three-dimensional elements in proteins [PDF]

, 2012
<br>Background: Protein structures incorporate characteristic three-dimensional elements defined by some or all of hydrogen bonding, dihedral angles and amino acid sequence.
Leader, D.P., Milner-White, E.J.
core   +3 more sources

Biomaterial Strategies for Targeted Intracellular Delivery to Phagocytes

Advanced Functional Materials, EarlyView.
Phagocytes are essential to a functional immune system, and their behavior defines disease outcomes. Engineered particles offer a strategic opportunity to target phagocytes, harnessing inflammatory modulation in disease. By tuning features like size, shape, and surface, these systems can modulate immune responses and improve targeted treatment for a ...
Kaitlyn E. Woodworth, Neal I. Callaghan, Locke Davenport Huyer   +2 more
wiley   +1 more source

DNA and its counterions: A molecular dynamics study [PDF]

, 2004
The behaviour of mobile counterions, Na+ and K+, was analysed around a B-DNA double helix with the sequence CCATGCGCTGAC in aqueous solution during two 50 ns long molecular dynamics trajectories.
Varnai, Peter, Zakrzewska, Krystyna
core   +3 more sources

Engineering Hybrid Extracellular Vesicles for Functional mRNA Delivery

Advanced Functional Materials, EarlyView.
This study optimizes two methods for the generation of extracellular vesicle (EV)‐mimetic‐Lipid Nanoparticle (LNP) (CELLNPs) and EV‐LNP (HEVs) hybrid nanoparticles for messenger (mRNA) delivery. The authors screen lipid compositions to maximize hybrid nanoparticle formation and analyze the resulting nanoparticles with single particle characterization ...
Ana Filipa Louro, Inês Gomes, Chuan‐En Lu, Marco A. Alfonzo‐Méndez, Niek Dekker, Mohsin Khan, Elisa Lázaro‐Ibáñez   +6 more
wiley   +1 more source