AI Article Synopsis
- Amyotrophic lateral sclerosis (ALS) is a deadly neurodegenerative disease that presents various clinical challenges and complex genetics, complicating understanding and treatment efforts.* -
- To address this, researchers created human induced pluripotent stem cells (iPSCs) from different ALS patients - including sporadic and familial cases - as well as healthy controls.* -
- These iPSCs can be used to develop 2D and 3D models of ALS, aiding in the study of disease mechanisms and the identification of potential therapies.*
Article Abstract
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease. Clinical heterogeneity and complex genetics pose challenges to understanding disease mechanisms and producing effective cures. To model clinical heterogeneity, we generated human induced pluripotent stem cells (iPSCs) from two sporadic ALS patients (sporadic ALS and sporadic ALS with frontotemporal dementia), two familial ALS patients (familial SOD1 mutation positive and familial C9orf72 repeat expansion positive), and four age- and sex-matched healthy controls. These iPSCs can be used to generate 2D and 3D in vitro models of ALS to investigate mechanisms of disease and screen for therapeutics.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1016/j.scr.2024.103447 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!