ALS research accelerated with new iPSC repository
The largest resource for amyotrophic lateral sclerosis (ALS) research has been made accessible to researchers worldwide.
Answer ALS (LA, USA) and Cedars-Sinai (CA, USA) have unveiled the largest repository of ALS patient-derived induced pluripotent stem cells (iPSCs) and associated biological data. This comprehensive collection includes clinical and biological data from nearly 1000 ALS patients, providing an invaluable resource for researchers worldwide studying ALS.
ALS is a progressive neurodegenerative disease that affects neurons in the brain and spinal cord. ALS mainly affects motor neurons, which are responsible for controlling voluntary muscle movement. As the condition progresses, these neurons deteriorate and die, causing progressive muscle weakness, loss of movement and, ultimately, the inability to breathe without support.
Research into the mechanisms behind ALS has largely involved genetically modified animal models in a variety of species, including fruit flies, zebrafish and rodents. However, animal models fail to fully capture the intricate complexity of human biology, especially given that over 40 genes have been associated with ALS.
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To address this challenge, Action ALS and Cedars-Sinai have worked together to develop a repository of patient-derived iPSCs that will offer an advanced model for disease research. Dhruv Sareen, Executive Director of the Cedars-Sinai Biomanufacturing Center, explained: “We reprogrammed blood cells into iPSCs and differentiated them into motor neurons, which die in ALS patients at an unprecedented scale.”
Along with the bank of patient-derived cells, the repository includes biological data such as patient-derived biofluids (e.g., blood or cerebrospinal fluid) and detailed clinical information on disease progression. Additionally, the repository offers multi-omics data, encompassing genomic, transcriptomic, proteomic, and epigenetic profiles. The data and associated bio-resources are accessible to the global research community via the Answer ALS Neuromine Data Portal and the Cedars-Sinai Biomanufacturing Center.
Clive Svendsen, Director of the Board of Governors Regenerative Medicine Institute at Cedars-Sinai, stated: “We believe this comprehensive platform will serve as a critical resource for the scientific community, propelling research into ALS pathogenesis, therapeutic development, and precision medicine approaches. The availability of this biorepository and the integration of a rich multi-omics dataset allows investigation of complex biological networks that drive ALS.”