Results 61 to 70 of about 25,568 (180)

More Than a Question of Correlation: Characterization of the Evidentiary Basis for Biomarker Surrogates Used in European Marketing Authorizations

Clinical Pharmacology &Therapeutics, EarlyView.
Traditionally, clinical outcomes measuring how a patient feels, functions, or survives are preferred endpoints in clinical trials; however, some may take a long time to manifest in slowly developing diseases. Biomarkers, if properly validated, can serve as surrogate endpoints, acting as substitutes for clinical outcomes.
Renske Johanna Grupstra, Viktoriia Starokozhko, Elisabeth Bakker, Anna Maria Gerdina Pasmooij, Peter Gerardus Maria Mol   +4 more
wiley   +1 more source

Engineering exosomal cargo loading via endogenous molecular pathways: Strategies to enhance therapeutic potential

Interdisciplinary Medicine, EarlyView.
This review illustrates how scientists engineer exosomes by hijacking the cell's own cargo‐sorting machinery. These strategies efficiently load therapeutic molecules into natural vesicles, creating powerful next‐generation drug delivery systems (Created with BioGDP.com).
Huanrong Zhu, Chenning Zhang, Shihan Ma, Zhanxue Xu, Cailing Liang, Fang Cheng, Hongbo Chen   +6 more
wiley   +1 more source

Chronic visceral acid sphingomyelinase deficiency (Niemann-Pick disease type B) in 16 Polish patients: long-term follow-up

Orphanet Journal of Rare Diseases, 2019
Background Acid sphingomyelinase deficiency (ASMD), due to mutations in the sphingomyelin phosphodiesterase 1 (SMPD1) gene, is divided into infantile neurovisceral ASMD (Niemann-Pick type A), chronic neurovisceral ASMD (intermediate form, Niemann-Pick ...
Patryk Lipiński, Ladislav Kuchar, Ekaterina Y. Zakharova, Galina V. Baydakova, Agnieszka Ługowska, Anna Tylki-Szymańska   +5 more
doaj   +1 more source

New amphiphilic amino acid derivatives for efficient DNA transfection in vitro [PDF]

, 2017
Nucleic acids-based therapies have recently developed as next-generationagents for treating and preventing viral infection, cancer, and genetic disorders,but their use is still limited due to its relatively poor delivery into targetedcells.
Antuña, Sebastián, Argarañá, María Fernanda, de Zan, María Mercedes, Giorello, Antonella, Muller, Diana, Peña, Lucía Carolina, Prieto, Claudio, Veaute, Carolina Melania Isabel   +7 more
core   +1 more source

Gene List Selection Matters: Missed Diagnoses in Prenatal Exome Sequencing—PanelApp R21 and HPO‐Driven Versus OMIM‐Based Gene Lists

Prenatal Diagnosis, EarlyView.
ABSTRACT Objective To evaluate whether the causative variants found upon clinical exome sequencing in fetuses affected with selected structural anomalies would also be detected if PanelApp‐R21 or Human Phenotype Ontology (HPO)‐driven gene selection terms were applied instead.
Victoria Ardiles‐Ruesjas, Laia Rodriguez‐Revenga, Montse Pauta, Alfons Nadal, Gemma Arca, Laura Gort, Stiven Ernesto Sinisterra‐Díaz, Antoni Borrell   +7 more
wiley   +1 more source

Boswellic acid inhibits expression of acid sphingomyelinase in intestinal cells

Lipids in Health and Disease, 2009
Background Boswellic acid is a type of triterpenoids with antiinflammatory and antiproliferative properties. Sphingomyelin metabolism generates multiple lipid signals affecting cell proliferation, inflammation, and apoptosis.
Duan Rui-Dong, Zhang Yao
doaj   +1 more source

Long-term biopermanence of ceramides, cholesteryl esters, and ether-linked triglycerides with very-long-chain PUFA in the cadmium-damaged testis [PDF]

, 2014
Cadmium is known to harm rat testis by causing the dose-dependent apoptotic or necrotic death of seminiferous epithelium cells. Here we investigated how this affects the lipids with long-chain (C18-C22) and very-long-chain (C24-C32) polyunsaturated fatty
Aveldaño, Marta I., Zanetti, Samanta Romina   +1 more
core   +2 more sources

A Curcumin‐Derived HSP70 Inhibitor Disrupts Lysosomal Function to Suppress Triple‐Negative Breast Cancer Progression

Cell Proliferation, EarlyView.
Novel curcumin‐derived M4 binds HSP70's ATPase domain, disrupting HSP70‐mediated lysosomes‐autophagy pathway to inhibit TNBC growth and metastasis, and synergizes with paclitaxel for potent combination therapy. ABSTRACT Structural modification of curcumin yielded a novel series of 1,4‐pentadien‐3‐one oxime ether derivatives, among which compound M4 ...
Zijian Li, Wanxia Wang, Yuxin Zhou, Tao Zeng, Jian Liu, Yingjie Qing, Xuan Han   +6 more
wiley   +1 more source

Holotoxin A1 Induces Apoptosis by Activating Acid Sphingomyelinase and Neutral Sphingomyelinase in K562 and Human Primary Leukemia Cells

Marine Drugs, 2018
Marine triterpene glycosides are attractive candidates for the development of anticancer agents. Holotoxin A1 is a triterpene glycoside found in the edible sea cucumber, Apostichopus (Stichopus) japonicus. We previously showed that cladoloside C2, the 25(
Seong-Hoon Yun, Eun-Hye Sim, Sang-Heum Han, Jin-Yeong Han, Sung-Hyun Kim, Alexandra S. Silchenko, Valentin A. Stonik, Joo-In Park   +7 more
doaj   +1 more source

Lysosomal cholesterol accumulation sensitizes to acetaminophen hepatotoxicity by impairing mitophagy.

, 2015
The role of lysosomes in acetaminophen (APAP) hepatotoxicity is poorly understood. Here, we investigated the impact of genetic and drug-induced lysosomal cholesterol (LC) accumulation in APAP hepatotoxicity.
Alarcón-Vila, C, Baulies, A, Fernández-Checa, JC, García-Ruiz, C, Kaplowitz, N, Martínez, L, Núñez, S, Ribas, V, Suda, J, Torres, S, Ybanez, MD   +10 more
core   +1 more source