Results 111 to 120 of about 156,351 (339)

The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease [PDF]

, 2022
Antonio Monaco, Veronica Maffia, Nicolina Cristina Sorrentino, Irene Sambri, Yulia Ezhova, Teresa Giuliano, Vincenzo Cacace, Edoardo Nusco, Maria De Risi, Elvira De Leonibus, Thomas Schräder, Frank‐Gerrit Klärner, Gal Bitan, Alessandro Fraldi   +13 more
openalex   +1 more source

Relative frequency and estimated minimal frequency of Lysosomal Storage Diseases in Brazil: Report from a Reference Laboratory

Genetics and Molecular Biology, 2017
Lysosomal storage diseases (LSDs) comprise a heterogeneous group of more than 50 genetic conditions of inborn errors of metabolism (IEM) caused by a defect in lysosomal function.
R. Giugliani, A. Federhen, Kristiane Michelin-Tirelli, M. Riegel, M. Burin   +4 more
semanticscholar   +1 more source

From RNA to DNA: How Cargo Identity Reprograms Lipid Nanoparticle Architecture and Function

Advanced Healthcare Materials, EarlyView.
The evolution of lipid nanoparticles (LNPs) spans from RNA‐LNPs, used in mRNA vaccines, to DNA‐LNPs, ideal for gene therapies. Emerging bionano architectures, decorated with DNA and plasma proteins, pave the way for advanced DNA‐based therapies that are more stable, targeted, and customizable.
Erica Quagliarini, Daniela Pozzi, Giulio Caracciolo   +2 more
wiley   +1 more source

Purification of the lysosomal sialic acid transporter. Functional characteristics of a monocarboxylate transporter [PDF]

, 1998
Sialic acid and glucuronic acid are monocarboxylated monosaccharides, which are normally present in sugar side chains of glycoproteins, glycolipids, and glycosaminoglycans.
Beerens, C.E.M.T. (Cecile), Havelaar, A.C. (Adrie), Mancini, G.M.S. (Grazia), Souren, R.M., Verheijen, F.W. (Frans)   +4 more
core   +4 more sources

Intrinsically Disordered Protein‐Inspired Nanovector‐Based Coacervates for the Direct Cytosolic Transport of Biomacromolecules

Advanced Materials, EarlyView.
Intrinsically disordered protein‐inspired nanovectors (IDP‐NVs) form stable nanocoacervates (NCs) with diverse biomacromolecules. In situ conformational changes confer stability and adaptability to NCs under dynamically changing physiological conditions.
Soyeong Jin, Hyemin Park, Seuk‐Min Ryu, Dagyeong Guk, Jaeeun Lee, Seongeon Jin, Changjoon Keum, Jinyoung Park, Myoung‐Hwan Park, Chaekyu Kim, Hojun Kim, Jaegeun Noh, Kwan Hyi Lee, Ja‐Hyoung Ryu, Youngdo Jeong   +14 more
wiley   +1 more source

Fundamentals of CNS energy metabolism and alterations in lysosomal storage diseases

Journal of Neurochemistry, 2018
The brain has a very high requirement for energy. Adult brain relies on glucose as an energy substrate, whereas developing brain can utilize alternative substrates as well as glucose for energy and for the biosynthesis of lipids and proteins required for
M. McKenna, P. F. Schuck, G. C. Ferreira
semanticscholar   +1 more source

Bacteria‐Responsive Nanostructured Drug Delivery Systems for Targeted Antimicrobial Therapy

Advanced Materials, EarlyView.
Bacteria‐responsive nanocarriers are designed to release antimicrobials only in the presence of infection‐specific cues. This selective activation ensures drug release precisely at the site of infection, avoiding premature or indiscriminate release, and enhancing efficacy.
Guillermo Landa, Gracia Mendoza, Silvia Irusta, Manuel Arruebo   +3 more
wiley   +1 more source

Pompe Disease: New Developments in an Old Lysosomal Storage Disorder

Biomolecules, 2020
Pompe disease, also known as glycogen storage disease type II, is caused by the lack or deficiency of a single enzyme, lysosomal acid alpha-glucosidase, leading to severe cardiac and skeletal muscle myopathy due to progressive accumulation of glycogen ...
Naresh K. Meena, Nina Raben
doaj   +1 more source

Recent Advances in Collective Behaviors of Micro/Nanomotor Swarms

Advanced Materials, EarlyView.
This review describes the driving forces behind collective motion, explores the self‐organization of micro/nano swarms across zero‐dimensional (0D), one‐dimensional (1D), two‐dimensional (2D), and three‐dimensional (3D) spaces, and highlights their potential in drug delivery, environmental monitoring, and smart devices.
Siwen Sun, Bingbing Sun, Alexander B. Cook, Tania Patiño Padial, Jan C. M. van Hest   +4 more
wiley   +1 more source

Reference values for lysosomal enzymes activities using dried blood spots samples - a Brazilian experience [PDF]

, 2010
Background: Lysosomal storage diseases (LSD) are inherited disorders caused by deficiency of lysosomal enzymes in which early diagnosis is essential to provide timely treatment.
Ana M Martins, Karen B Müller, Mayra DB Rodrigues, Vanessa G Pereira, Vânia D'Almeida   +4 more
core   +2 more sources