Results 41 to 50 of about 27,697 (237)

Prevalence of Lysosomal Storage Disorders [PDF]

JAMA, 1999
Lysosomal storage disorders represent a group of at least 41 genetically distinct, biochemically related, inherited diseases. Individually, these disorders are considered rare, although high prevalence values have been reported in some populations. These disorders are devastating for individuals and their families and result in considerable use of ...
Meikle, P., Hopwood, J., Clague, A., Carey, W.   +3 more
openaire   +3 more sources

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

Lyso-IP: Uncovering Pathogenic Mechanisms of Lysosomal Dysfunction

Biomolecules, 2022
Lysosomes are ubiquitous membrane-bound organelles found in all eukaryotic cells. Outside of their well-known degradative function, lysosomes are integral in maintaining cellular homeostasis. Growing evidence has shown that lysosomal dysfunction plays an
Chase Chen, Ellen Sidransky, Yu Chen
doaj   +1 more source

Nanotherapies for Atherosclerosis: Targeting, Catalysis, and Energy Transduction

Advanced Healthcare Materials, EarlyView.
Atherosclerosis management is hindered by poor drug targeting and plaque heterogeneity. Nanotechnology overcomes these barriers via three core strategies: (1) target‐engineered nanocarriers that achieve lesion‐specific precision via ligand modification, biomimetic camouflage, stimuli‐responsive release, and self‐propelling nanomotors; (2) catalytic ...
Yuqi Yang, Jingguo Qu, Tonghuan Xiao, Xinyang Han, Michael Tin‐Cheung Ying   +4 more
wiley   +1 more source

Rethinking Extracellular Vesicle Signaling

Advanced Materials, EarlyView.
Extracellular vesicles enable cell communication beyond intracellular cargo delivery. This perspective highlights two plausible surface‐based signaling modes: “bind‐and‐stay” and “bind‐and‐leave.” Transient binding to multiple cells challenges the one‐vesicle‐one‐cell model.
Wojciech Chrzanowski, Joy Wolfram
wiley   +1 more source

Radiotherapy Enhancement by Gold Nanocluster‐functionalized Nanoliposomes Using Polychromatic Orthovoltage X‐ray Irradiation

Advanced Materials Interfaces, EarlyView.
Lipid drug carriers to which ultra‐small gold nanoparticles were added enabled more efficient radiotherapy of cultured pancreatic cancer tumors. These nanoparticles boosted radiation‐induced damage to tumors by generating more reactive molecules, though higher gold levels are needed for strong benefits.
Nazareth Milagros Carigga Gutierrez, Eaazhisai Kandiah, Sofia Leo, Erwan Dupont, Fabrizio Johnson‐Corrales, Marc‐André Hograindleur, Sarvenaz Keshmiri, Hélène Elleaume, Benoit Busser, Romain Haudecoeur, Véronique Jacob, Anthony Vella, Véronique Josserand, Maxime Henry, Jean‐Luc Coll, Amandine Hurbin, Xavier Le Guével, Mans Broekgaarden   +17 more
wiley   +1 more source

Safety analysis of self-administered enzyme replacement therapy using data from the Fabry Outcome and Gaucher Outcome Surveys

Orphanet Journal of Rare Diseases
Background Fabry disease and Gaucher disease are rare genetic disorders characterized by defective degradation of glycosphingolipids caused by enzymatic deficiencies in α–galactosidase A and β–glucocerebrosidase, respectively, and often require life-long
Shoshana Revel-Vilk, Uma Ramaswami, Guillem Pintos-Morell, Derralynn Hughes, Kathy Nicholls, Ricardo Reisin, Roberto Giugliani, Ozlem Goker-Alpan, Majdolen Istaiti, Aidan Gill, Maurizio Scarpa, Jaco Botha   +11 more
doaj   +1 more source

Phenotype Correction in Murine Mucopolysaccharidosis Type VII by Transplantation of Human Amniotic Epithelial Cells after Adenovirus-Mediated Gene Transfer

Cell Transplantation, 2000
Cell therapy with human amniotic epithelial (HAE) cells was developed as an alternative method for enzyme replacement therapy in congenital lysosomal storage disorders, but only limited therapeutic efficacy has been reported.
Motomichi Kosuga, Satori Takahashi, Kyoko Sasaki, Shin Enosawa, Xiao-Kang Li, Sanae Okuyama, Masayuki Fujino, Seiichi Suzuki, Masao Yamada, Nobutake Matsuo, Norio Sakuragawa, Torayuki Okuyama   +11 more
doaj   +1 more source

Differentiation of norm and pathology during selective biochemical skreening of lysosomal storage diseases with increased excretion of oligosaccharides [PDF]

The Ukrainian Biochemical Journal, 2015
Oligosaccharides are a class of polymeric carbohydrates, which are constituents of a glycoside portion of glycoprotein and glycolipid molecules. The lysosomal hydrolase dysfunction due to lysosomal storage disorders results in partial or complete failure
N. Y. Mytsyk, N. V. Olkhovych, N. G. Gorovenko   +2 more
doaj   +1 more source

Multimodal Actuation and Environment Adaptive Strategies of Bio‐Inspired Micro/Nanorobots in Precision Medicine

Advanced Robotics Research, EarlyView.
An introduction for multidrive and environment‐adaptive micro/nanorobotics: design and fabrication strategies, intelligent actuation, and their applications. Various intelligent actuation approaches—magnetic, acoustic, optical, chemical, and biological—can be synergistically designed to enhance flexibility and adaptive behavior for precision medicine ...
Aiqing Ma, Han Gong, Jiacheng Ouyang, Liqi Chen, Tianfang Kang, Yiran He, Guojun Huang, Sidi Zheng, Ting Yin, Hong Pan, Lintao Cai   +10 more
wiley   +1 more source