Results 111 to 120 of about 36,163 (246)

Gene therapy for lysosomal storage diseases: Current clinical trial prospects. [PDF]

Front Genet, 2023
Kido J, Sugawara K, Nakamura K.
europepmc   +1 more source

Dynamic Shifts in ER–Plasma Membrane Junctions Signaling Define Pro‐Metastatic N‐Glycosylation and Predict Prostate Cancer Progression

Advanced Science, EarlyView.
Prostate cancer remains a leading cause of male cancer death, yet screening cannot reliably identify aggressive disease, underscoring the need for tissue biomarkers. It is shown that primary tumors increase ER–plasma membrane junction signaling via STIM1/ORP5, whereas metastasis features their loss, Golgi dispersal, and rapid conversion of high‐mannose
Amanda J. Macke, Tania Kamal, Taylor E. Divita, Artem N. Pachikov, Chad A. LaGrange, Rajesh Ravichandran, Martha Morton, Robert Powers, Haowen Qiu, Jean‐Jack M. Riethoven, Colm Morrissey, Melinda Wojtkiewicz, Rebekah L. Gundry, Carol A. Casey, Armen Petrosyan   +14 more
wiley   +1 more source

NIR‐II Imaging‐Guided Photothermal Activation of a TRPV4‐Targeted Nanoplatform Delivering Cycloastragenol to Promote Microglia Reprogramming and α‐Synuclein Clearance in Parkinson's Disease

Advanced Science, EarlyView.
ABSTRACT Current therapies for Parkinson's disease (PD) fail to concurrently address α‐synuclein (α‐syn) aggregation and microglia‐mediated neuroinflammation. Herein, we engineer a near‐infrared‐II (NIR‐II) phototheranostic nanoplatform, CAG/FD1080@MM‐aTRPV4, for synergistic regulation of microglial function and real‐time monitoring of PD pathology. We
Hsuan Lo, Linjuan Feng, Shiying Li, Lik Hang Hugo Tse, Xuehan Wang, Xingyang Zhao, Shaoheng Ma, Xin Li, Yanjuan Gu, Wing‐Tak Wong   +9 more
wiley   +1 more source

Redefining the Health Risk of Battery Materials Through a Biologically Transformed Metal Mixture

Advanced Science, EarlyView.
Inhaled NCM particles undergo lysosomal degradation, releasing complex ion mixtures that induce systemic impact. The impact is determined by a critical balance between antagonistic Ni‐Co interactions and synergistic Mn effects. To capture these complexities in risk assessment, we develop an IAI model, ensuring a more accurate quantitative risk ...
Ze Zhang, Gan Miao, Xueyu Zhang, Zhao Shu, Dawei Lu, Yujie Song, Shanfa Yu, Qian Liu, Yang Song, Rong Zhang, Xiaoting Jin, Yuxin Zheng   +11 more
wiley   +1 more source

Tumor‐Induced Splenic Remodeling: Mechanisms of Systemic Immunosuppression and Emerging Therapeutic Opportunities

Advanced Science, EarlyView.
ABSTRACT Despite the transformative impact of cancer immunotherapies such as immune checkpoint blockade, durable clinical responses remain limited. Increasing evidence indicates that antitumor immunity is governed not only by the tumor microenvironment, but also by systemic immune regulation mediated by peripheral immune organs. Among these, the spleen
Yuehua Liu, Xiaoqian Nie, Xiaofei Gao
wiley   +1 more source

Novel Fused Pyrazole Amide Analogs as Glucosylceramide Synthase Inhibitors for Treating Lysosomal Storage Diseases, Neurodegenerative Diseases, Cystic Diseases, and Cancer. [PDF]

ACS Med Chem Lett, 2023
Sabnis RW.
europepmc   +1 more source

Nanozymes for Liver Disease Therapy: Advances in Catalytic Activity, Targeting Strategies, and Clinical Translation

Advanced Science, EarlyView.
Nanozymes, as enzyme‐mimicking nanomaterials, exhibit unique catalytic properties for the treatment of liver diseases. By regulating redox homeostasis, modulating immune responses, and enabling targeted delivery, nanozymes overcome the limitations of natural enzymes.
Xiandi Meng, Ge Zhu, Siyu Sun, Wenbo Yao, Yuning Zhang, Yong‐Guang Yang, Tianmeng Sun   +6 more
wiley   +1 more source

Human, economic, and social impact of lysosomal storage diseases. [PDF]

Orphanet J Rare Dis
Brignani E, de Castro-López MJ, Gonzalez-Meneses A, Morales M, Pintos-Morell G, Poveda JL, Vilalta I, Cruz J, Ballester-Lozano GF, David de Paz H.   +9 more
europepmc   +1 more source

Unveiling a New Link: Cholesterol Deficiency in Smith–Lemli–Opitz and Niemann–Pick C as a Driver of Ciliopathies

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT The ciliopathies are a group of genetic disorders caused by defective function of either the primary cilia (a large number) or the motile cilia (a much smaller number). These have been defined as diseases with mutations in genes encoding individual ciliary or cilia‐associated proteins.
Robert P. Erickson, Maria Teresa Fiorenza   +1 more
wiley   +1 more source

Nonimmune Hydrops Fetalis and Lysosomal Storage Diseases

Pediatrics and Neonatology, 2013
Carlo Bellini
doaj   +1 more source