Results 131 to 140 of about 123,112 (260)

Dried Blood Spot (DBS) Methodology Study for Biomarker Discovery in Lysosomal Storage Disease (LSD). [PDF]

Metabolites, 2021
Rus CM, Di Bucchianico S, Cozma C, Zimmermann R, Bauer P.   +4 more
europepmc   +1 more source

The Immune Microenvironment in Liver Cancer: From Analysis to Targeting

Advanced Science, EarlyView.
This review explores how smart nanomedicines overcome the immunosuppressive tumor microenvironment in liver cancer. It details targeted delivery strategies and immune reprogramming mechanisms, including remodeling abnormal physiology, modulating metabolism, and inducing immunogenic cell death. The article highlights the paradigm shift toward multimodal
Jiaming Lan, Hao Li, Jian Xue, Yourong Duan, Jin Sun, Meng Niu   +5 more
wiley   +1 more source

Prevalence and management of Gaucher disease

Pediatric Health, Medicine and Therapeutics, 2011
T Andrew Burrow, Sonya Barnes, Gregory A GrabowskiThe Division of Human Genetics, Cincinnati Children's Hospital Medical Center, and the Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USAAbstract: Gaucher ...
Burrow TA, Barnes S, Grabowski GA
doaj  

Advances in miRNA research: Unraveling the complexities of gene regulation

Animal Models and Experimental Medicine, EarlyView.
Overview of miRNA‐mediated regulation in key biological processes. This illustration offers a comprehensive view of the diverse functions that microRNAs perform in governing various biological processes, highlighting their profound significance within the complex web of gene expression and cellular function.
Jiawei Zheng, Guoqing Zhang, Linzhu Ren
wiley   +1 more source

PDB123 CURRENT STATUS AND USE OF RESOURCES OF LYSOSOMAL STORAGE DISEASES: ANALYSIS OF A SPANISH CLAIMS DATABASE [PDF]

, 2019
Josep Darbà, M. Ascanio, Alícia Marsà
openalex   +1 more source

Construction of pathogenic Sec16a mutation mouse model using CRISPR/Cas9

Animal Models and Experimental Medicine, EarlyView.
Yaqiang Hu et al. engineered a pathogenic Sec16a mutant mouse model using CRISPR/Cas9 technology. They observed that the Sec16a mutant mice displayed diminished learning and memory capabilities, along with a limb‐clasping phenotype upon tail suspension.
Yaqiang Hu, Zhiyang Zeng, Xinyu Ming, Shuming Yin, Yuting Guan, Liangcai Gao, Dali Li   +6 more
wiley   +1 more source

Examining the Role of a Functional Deficiency of Iron in Lysosomal Storage Disorders with Translational Relevance to Alzheimer’s Disease [PDF]

, 2023
Steven M. LeVine
openalex   +1 more source

N‐Amino Peptide–Graphene Quantum Dot Loaded Small Extracellular Vesicles for Targeted Therapy of Tauopathies

Advanced NanoBiomed Research, EarlyView.
A neuron‐targeting drug delivery system is developed by incorporating the tau‐specific N‐amino peptide, mxyl‐NAP2, with graphene quantum dots (GQDs) and encapsulating the complex into small extracellular vesicles functionalized with a neuron‐targeting ligand.
Runyao Zhu, Gaeun Kim, Benjamin H. Rajewski, Isaac J. Angera, Juan R. Del Valle, Yichun Wang   +5 more
wiley   +1 more source

Inhibition of substrate synthesis as a strategy for glycolipid lysosomal storage disease therapy

, 2001
Frances M. Platt, Mylvaganam Jeyakumar, Ulrika Andersson‐Hall, David A. Priestman, Raymond A. Dwek, Terry D. Butters, Timothy M. Cox, Robin Lachmann, Carla E. M. Hollak, Johannes M. F. G. Aerts, Sonja van Weely, Martin Hřebı́ček, Chris Moyses, Iain F Gow, Deborah Elstein, Ari Zimran   +15 more
openalex   +2 more sources

Cell line construction and maintenance for Lyso-IP with or without genes linked with lysosomal storage disease v1 [PDF]

, 2022
Sharan Sharan Swarup
openalex   +1 more source