Category Ranking
98%
Total Visits
921
Avg Visit Duration
2 minutes
Citations
20
Article Abstract
Amyotrophic lateral sclerosis (ALS) is a fatal neurological disorder characterized by progressive degeneration of nerve cells in the spinal cord and brain. We generated and characterized a human induced pluripotent stem cell (iPSC) line from skin fibroblasts of a patient with ALS due to SOD1 Mutation. The pluripotency of these iPSCs was verified by the expression of several pluripotency markers at both RNA and protein levels, as well as their capability to differentiate into all three germ layers.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1016/j.scr.2025.103704 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Familial Alzheimer's disease mutation identifies novel role of SORLA in release of neurotrophic exosomes.
Alzheimers Dement
September 2025
Department of Biomedicine, Aarhus University, Aarhus, Denmark.
Introduction: Mutations in SORL1, encoding the sorting receptor Sortilin-related receptor with A-type repeats (SORLA), are found in individuals with Alzheimer's disease (AD). We studied SORLA, carrying a mutation in its ligand binding domain, to learn more about receptor functions relevant for human brain health.
Methods: We investigated consequences of SORLA expression in induced pluripotent stem cell (iPSC)-derived human neurons and microglia, using unbiased proteome screens and functional cell assays.
iPSC-hepatocyte organoids as a novel platform to predict AAV gene therapy efficacy.
Mol Ther Methods Clin Dev
June 2025
Precision Safety, Pharma Product Development, Roche Innovation Center Basel, CH-4070 Basel, Switzerland.
Adeno-associated virus (AAV) vectors are widely used in gene therapy, particularly for liver-targeted treatments. However, predicting human-specific outcomes, such as transduction efficiency and hepatotoxicity, remains challenging. Reliable models are urgently needed to bridge the gap between preclinical studies and clinical applications.
View Article and Find Full Text PDFEstablishment and optimization of the two-step induction system for generating primordial germ cell-like cells from chicken embryonic stem cells.
FEBS Open Bio
September 2025
Joint International Research Laboratory of Agriculture and Agri-Product Safety of Ministry of Education of China, Yangzhou University, China.
Primordial germ cells (PGCs) are the progenitor cells of sperm and eggs. Xenotransplantation of chicken PGCs can achieve germline transmission. However, there are still challenges in obtaining many PGCs from endangered birds in vitro.
View Article and Find Full Text PDFCETN3 deficiency induces microcephaly by disrupting neural stem/progenitor cell fate through impaired centrosome assembly and RNA splicing.
EMBO Mol Med
September 2025
Institute for Regenerative Medicine, Medical Innovation Center and State Key Laboratory of Cardiovascular Diseases, Shanghai East Hospital, National Stem Cell Translational Resource Center & Ministry of Education Stem Cell Resource Center, Frontier Science Center for Stem Cell Research, School of Li
Primary microcephaly, a rare congenital condition characterized by reduced brain size, occurs due to impaired neurogenesis during brain development. Through whole-exome sequencing, we identified compound heterozygous loss-of-function mutations in CENTRIN 3 (CETN3) in a 5-year-old patient with primary microcephaly. As CETN3 has not been previously linked to microcephaly, we investigated its potential function in neurodevelopment in human pluripotent stem cell-derived cerebral organoids.
View Article and Find Full Text PDFValidating and Using Cardiac NAMs for Toxicity Screening and Drug Development.
Int J Toxicol
September 2025
RTI International, Washington, DC, USA.
Technological advances and the desire to reduce dependence on animal models have brought human-relevant models to the forefront of drug development. This paradigm shift is leveraging the advances in systems and new approach methodologies (NAMs), which was the focus of a workshop convened by the Health and Environmental Sciences Institute (HESI) in May 2024. Highlights included discussions on predicting cardiac failure modes and the utility of human-induced pluripotent stem cell-derived cardiomyocytes (hiPSC-CMs), microfluidic systems like BioFlux™, and engineered heart tissues in enhancing early-stage drug safety assessments.
View Article and Find Full Text PDF