Target ALS Announces Expansion of Stem Cell Resources to Accelerate ALS Research
August 11, 2026 Amy Easton
Target ALS is proud to announce a powerful new resource for ALS researchers: a collection of 28 standardized, well-characterized, and accessible fibroblast-derived induced pluripotent stem cell (iPSC) lines, available through the Stem Cell Core, with no strings attached. The lines represent key ALS-associated genetic variants and are each paired with an isogenic control. Both academic and industry researchers can now access these lines directly through The Jackson Laboratory.
Human iPSC-derived models are critical tools for studying ALS biology and testing candidate therapeutics, as they can uniquely capture human disease mechanisms that animal systems cannot. However, generating and characterizing iPSC lines is resource-intensive and technically challenging for any single lab. Reproducibility has been difficult to achieve given the use of different protocols and the lack of uniform human iPSC-line accessibility across academia and industry groups. Further, logistical and financial barriers have long prevented scientists from leveraging available models.
To address these challenges, in 2024, Target ALS assembled a consortium of iPSC experts. This resulting collection provides standardized, well-characterized iPSC lines to advance disease modeling and drug discovery efforts, available with no-strings-attached to global scientists focused on ALS.
About the Collection
The collection includes seven patient-derived lines and matching CRISPR-corrected isogenic control lines spanning four of the most well-studied ALS-associated genes:
- C9ORF72 (n=2)
- TARDBP (n=1)
- FUS (n=2)
- SOD1 (n=2)
These same seven patient-derived lines and matching isogenic control lines may also be requested with a stably integrated, doxycycline-inducible NGN2 knock-in to enable rapid, robust, and reproducible differentiation into neurons.
- C9ORF72 (n=2)
- TARDBP (n=1)
- FUS (n=2)
- SOD1 (n=2)
Every line has undergone extensive quality control and phenotypic characterization and has been tested across multiple labs to assess reproducibility of ALS phenotypes. These isogenic pairs allow scientists to study the mutation on the same genetic background and therefore attribute cellular phenotypes specific to the mutation of interest.
Lines will be listed under Donor-Derived iPSCs / Target ALS Collection in the JAX iPSC catalog.
Democratizing Access to Critical Research Resources
In addition, linked genomic, transcriptomic, and clinical data, including long-read sequencing data that characterizes repeat expansions and other complex genomic features generated with PacBio, will be available through the Target ALS Data Engine.
Critically, this resource has been designed for maximum accessibility: the lines are appropriately consented for broad use, carry no licensing fees, and are available to both academic and industry researchers through the Jackson Laboratory. By removing the financial and logistical barriers that have historically limited iPSC-based research, this new collection stands to accelerate reproducible, standardized ALS disease modeling across the global research community.
A Collaborative Effort
This resource was developed through the collaborative efforts of Barbara Corneo, PhD (Director of the Stem Cell Core, Columbia University), and Bill Skarnes, PhD (The Jackson Laboratory), who led the creation of cell lines.
Consortium members who contributed to this effort include:
- Sami Barmada, MD, PhD (University of Michigan)
- Johnathan Cooper-Knock, PhD (University of Sheffield)
- Eran Hornstein, PhD (Weizmann Institute)
- Jimena Andreas, PhD (Emory University)
- João Duarte Tavares de Silva Pereira, PhD (Yale University)
By bringing together leading experts to create this shared resource, the Target ALS Stem Cell Consortium is helping to strengthen reproducibility, reduce barriers to research, and accelerate discovery across the ALS research community.
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