Results 71 to 80 of about 39,202 (243)

The Link Between Lysosomal Storage Disorders and More Common Diseases

Journal of Inborn Errors of Metabolism and Screening, 2016
In the last decades, it has become more and more evident that lysosomal storage disorders and common neurodegenerative diseases such as Alzheimer and Parkinson diseases have clinical, neuropathological, and genetic features in common, including lysosomal
Michael Beck MD
doaj   +1 more source

Lysosomal exocytosis and lipid storage disorders

Journal of Lipid Research, 2014
Lysosomes are acidic compartments in mammalian cells that are primarily responsible for the breakdown of endocytic and autophagic substrates such as membranes, proteins, and lipids into their basic building blocks. Lysosomal storage diseases (LSDs) are a
Mohammad Ali Samie, Haoxing Xu
doaj   +1 more source

Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases

Frontiers in Molecular Biosciences, 2020
There are over 50 lysosomal hydrolase deficiencies, many of which cause neurodegeneration, cognitive decline and death. In recent years, a number of broad innovative therapies have been proposed and investigated for lysosomal storage diseases (LSDs ...
Jacob M. Favret, Nadav I. Weinstock, M. Laura Feltri, Daesung Shin   +3 more
doaj   +1 more source

Compensatory Mitophagy via NEDD4/HIF‐1α/BNIP3 Pathway Restrains EndMT and Renal Allograft Interstitial Fibrosis Induced by TNFα

Advanced Science, EarlyView.
The role of TNFα in the process of renal allograft interstitial fibrosis is complex and multifaceted. As it can promote renal allograft interstitial fibrosis by inducing mitochondrial dysfunction and EndMT, and also mediating compensatory mitophagy through the NEDD4–HIF‐1α–BNIP3 pathway, which clears damaged mitochondria and inhibits EndMT, thereby ...
Dengyuan Feng, Qinghuan Shen, Junqi Zhang, Jiawen Liu, Zeping Gui, Zijie Wang, Qianguang Han, Shuang Fei, Hao Chen, Li Sun, Jun Tao, Zhijian Han, Xiaobing Ju, Min Gu, Jianjian Zhang, Ruoyun Tan   +15 more
wiley   +1 more source

Mr BMT Achieves Systemic Macrophage Replacement With Preservation of Tissue Homeostasis

Advanced Science, EarlyView.
Microglia replacement by bone marrow transplantation (Mr BMT) enables systemic replacement of tissue‐resident macrophages. Despite persistent macrophage and tissue remodeling across multiple organs, core biological functions and innate immune responses remain preserved, supporting long‐term maintenance of organismal homeostasis and the therapeutic ...
Yufei Xu, Miaozhan Zou, Yunshang Bai, Yang He, Zhihao Jin, Baozhi Yang, Jinjin Du, Pei Ouyang, Yuanyuan Cai, Yuheng Zhang, Bingying Du, Chengcheng Ge, Jian‐an Gong, Xiaoyu Li, Yuanyi Zheng, Jing Ding, Bo Peng, Yanxia Rao   +17 more
wiley   +1 more source

Innate Immunocompetent hiPSC‐Derived Neurospheroids Capture Early CNS Responses to rAAV

Advanced Science, EarlyView.
Knowledge of human CNS immune responses to AAV‐based gene therapies remains limited due to the lack of immune‐competent human models. Here, a hiPSC‐derived 3D neuroimmune platform integrating neurospheroids and microglia is established using stirred‐tank bioreactors.
Catarina M. Gomes, Gabriela Silva, Mafalda Aleixo, Marta Gomes, Daniel Simão, Stephan J. Holtkamp, Diana D. Lobo, Pradeep Harish, Rosalind Jenkins, Lekh N. Dahal, Rui J. Nobre, Luís de Pereira de Almeida, Mark Trautwein, Paula M. Alves, Catarina Brito   +14 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

Charting Endocrine Progenitors Across Species and Organs

Advanced Science, EarlyView.
Endocrine progenitors give rise to the hormone‐producing cells of the pancreas and intestine. Using single‐cell multiomics and proteomics, this study compares these progenitors across species, systems, and organs, mapping the conserved and species‐specific gene regulatory networks that guide their formation.
Changying Jing, Michael Sterr, Özüm Sehnaz Caliskan, Lama Saber, Nicole Katarina Rogers, Samuel Ogden, Kei Kozawa, Christos Karampelias, Jessica Jaki, Aimée Bastidas‐Ponce, Falk Janos Farkas, Oliver Czarnecki, Osvaldo Rodrigues Pereira, Perla Cota, Valerie Vandenbempt, Diego Balboa, Anika Böttcher, Natalie Krahmer, Mohammad Lotfollahi, Julie B. Sneddon, Heiko Lickert, Mostafa Bakhti   +21 more
wiley   +1 more source

Ultrasound‐Activatable Piezoelectric Hydrogel Reprograms Mitochondrial Epigenetics for Osteoarthritis Therapy via the mTOR/GATD3A Axis

Advanced Science, EarlyView.
An ultrasound‐activatable piezoelectric hydrogel reprograms chondrocyte mitochondrial epigenetics via the mTOR/GATD3A axis, clearing damaged mitochondria and alleviating osteoarthritis progression in both mouse models and human cartilage explants. ABSTRACT The avascular nature of cartilage hinders drug delivery for osteoarthritis (OA) therapy.
Hui Zheng, Pengfei Yan, Peng Liu, Chang Yan, Mengqi Zhao, Zuyong Wang, Chao Ma, Rongkai Zhang, Xi Cui, Swee Hin Teoh   +9 more
wiley   +1 more source

AI‐Assisted Engineering of Glycyrrhizic Acid/Simvastatin Nanocrystals for Multifunctional Treatment of Bacterial Osteomyelitis

Advanced Science, EarlyView.
Through AI‐assisted screening from FDA‐approved API to overcome the limitations of bacterial osteomyelitis treatment, glycyrrhizic acid and simvastatin are identified as a multifunctional combination capable of self‐assembling into mechanism‐targeting nanocrystals that effectively neutralize reactive oxygen species, suppress M1 macrophage polarization,
Yu Han, Yao Zhao, Chengbo Yang, Miao Niu, Jun Dai, Quanxin Ning, Kai Xiao, Jinzheng Liang, Wensheng Zhang, Liang Wang, Dan Shao, Dongsong Li   +11 more
wiley   +1 more source