Results 81 to 90 of about 21,042 (237)

Modest phenotypic improvements in ASA-deficient mice with only one UDP-galactose:ceramide-galactosyltransferase gene

Lipids in Health and Disease, 2006
Summary Background Arylsulfatase A (ASA)-deficient mice are a model for the lysosomal storage disorder metachromatic leukodystrophy. This lipidosis is characterised by the lysosomal accumulation of the sphingolipid sulfatide.
De Deyn PP, D'Hooge R, Mansson JE, Wittke D, Franken S, Lüllmann-Rauch R, Matzner U, Gieselmann V   +7 more
doaj   +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

Extracellular Vesicles Released by Genetically Modified Macrophages Activate Autophagy and Produce Potent Neuroprotection in Mouse Model of Lysosomal Storage Disorder, Batten Disease. [PDF]

Cells, 2023
El-Hage N, Haney MJ, Zhao Y, Rodriguez M, Wu Z, Liu M, Swain CJ, Yuan H, Batrakova EV.   +8 more
europepmc   +1 more source

Arylsulfatase A deficiency causes seminolipid accumulation and a lysosomal storage disorder in Sertoli cells [PDF]

, 2011
Hongbin Xu, Kessiri Kongmanas, Suraj Kadunganattil, Charles E. Smith, Tony Rupar, Naoko Goto‐Inoue, Louis Hermo, Kym F. Faull, Nongnuj Tanphaichitr   +8 more
openalex   +1 more source

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

The Association Between Lysosomal Storage Disorder Genes and Parkinson's Disease: A Large Cohort Study in Chinese Mainland Population. [PDF]

Front Aging Neurosci, 2021
Zhao YW, Pan HX, Liu Z, Wang Y, Zeng Q, Fang ZH, Luo TF, Xu K, Wang Z, Zhou X, He R, Li B, Zhao G, Xu Q, Sun QY, Yan XX, Tan JQ, Li JC, Guo JF, Tang BS.   +19 more
europepmc   +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

Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic. [PDF]

Cell, 2021
Logan T, Simon MJ, Rana A, Cherf GM, Srivastava A, Davis SS, Low RLY, Chiu CL, Fang M, Huang F, Bhalla A, Llapashtica C, Prorok R, Pizzo ME, Calvert MEK, Sun EW, Hsiao-Nakamoto J, Rajendra Y, Lexa KW, Srivastava DB, van Lengerich B, Wang J, Robles-Colmenares Y, Kim DJ, Duque J, Lenser M, Earr TK, Nguyen H, Chau R, Tsogtbaatar B, Ravi R, Skuja LL, Solanoy H, Rosen HJ, Boeve BF, Boxer AL, Heuer HW, Dennis MS, Kariolis MS, Monroe KM, Przybyla L, Sanchez PE, Meisner R, Diaz D, Henne KR, Watts RJ, Henry AG, Gunasekaran K, Astarita G, Suh JH, Lewcock JW, DeVos SL, Di Paolo G.   +52 more
europepmc   +1 more source

A perspective on research, diagnosis, and management of lysosomal storage disorders in Colombia [PDF]

, 2020
María Alejandra Puentes-Tellez, Paula Andrea Lerma-Barbosa, Rafael Guillermo Garzón-Jaramillo, Diego A. Súarez, Ángela J. Espejo, Johana Guevara, Olga Yaneth Echeverri-Peña, Daniela Solano-Galarza, Alfredo Uribe, Carlos Javier Alméciga-Díaz   +9 more
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