Stem Cell Core
A growing repository of well-characterized iPSC ALS and control lines, including isogenic and reporter lines, generated by leading partners and stored and managed by Sampled.
How we’re unlocking human models of ALS
Human iPSC-derived models are now critical tools for studying ALS and FTD biology and for testing candidate therapeutics. These models can uniquely capture human disease mechanisms that animal systems cannot. But generating and characterizing iPSC lines is resource-intensive and technically challenging for any single lab.
The Target ALS Stem Cell Core makes this technology broadly accessible to both academia and industry, with no reach-through on data or intellectual property.
What’s available through the Stem Cell Core?
iPSC lines created through partnerships with Columbia University Irving Medical Center, catalogued in the NINDS repository managed by Sampled. These include:
- iPSCs from C9orf72, SOD1, and TDP43 mutation carriers and mutation-negative iPSCs
- iPSCs from healthy individuals
- iPSC reporter lines created by Corneo lab and incorporating GFP or tdTomato TakaRa fluorescent proteins
- Associated clinical and genomic datasets
All lines were generated from Dermal fibroblasts from skin biopsies using a Sendai-virus based approach.
View publications and protocols to guide use of these lines:
What’s in development in the Stem Cell Core
Through the Stem Cell Core Consortium, we are expanding the collection with well-characterized patient-derived iPSCs carrying key ALS mutations:
- C9ORF72 (n=2)
- TARDBP (n=2)
- FUS (n=2)
- SOD1 (n=2)
Each line will be paired with a CRISPR-corrected control and validated across multiple labs to test reproducibility. This effort addresses variability challenges across labs, genetic backgrounds, and even sex, ensuring robust, reproducible phenotypes for the field:
- Patient and CRISPR-corrected isogenic control lines to enable precise disease vs. control comparisons.
- Doxycycline-inducible activation of NGN2 inserted at safe-harbor loci for reproducible differentiation into motor neurons.
- QC and phenotypic characterization of lines including linked genomic and clinical data available through the [Target ALS Data Engine].
Consortium members include:
- Sami Barmada, MD, PhD (University of Michigan)
- Johnathon Cooper-Knock, PhD (University of Sheffield)
- Eran Hornstein, PhD (Weizmann Institute)
- Jimena Andreas, PhD (Emory University)
- Joao Duarte Tavares de Silva Pereira, PhD (Yale University)
Cell line creation:
- Bill Skarnes, PhD (The Jackson Laboratory)
- Barbara Corneo, PhD (Columbia University)
Expected availability of first lines: Early 2026
Why Target ALS’s Stem Cell Core matters
Target ALS provides centralized access to stem cell lines, differentiation protocols, and genomic data. Well-characterized lines provide researchers confidence in the material they request, reduces time to get important experiments up and running, and allows for better science. All lines that can be used for research purposes by industry and academic organizations.
Researchers should check MTAs for lines where specific licenses may be needed to do research with the line.
The benefits of our Stem Cell Core:
- Cross-group validation of phenotype reproducibility.
- Integrated data through the Target ALS Data Engine.
- Accelerated discovery with tools designed for scalability, including high-throughput screening.
