The Target ALS Stem Cell Core is a network of contract research organizations and academic core facilities that generate and distribute patient-derived stem cell lines and associated data to investigate underlying disease mechanisms and the effect of drugs on disease pathology in ALS. Lines come from healthy individuals and from patients with sporadic or familial forms of ALS including C9orf72, SOD1, and TDP43 mutations. WGS data for each line is available in the portal. RNAseq data exists for a subset of the lines. In 2025, several new patient lines will become available. They will be edited with knockin of transcription factors to facilitate differentiation into neurons. Matching isogenic control lines, crispr-edited to correct the patient mutation will also be available. Data from these iPSC lines, including WGS, RNAseq, and proteomics, will be available for analysis. Learn more about the Stem Cell Core at https://www.targetals.org/resource/stem-cell-core/ Data Dictionaries Data Dictionaries for Stem Cell Core Tables Methods NYGC Germline Analysis Pipeline NYGC Short-Read RNA Sequencing NYGC Short-Read Whole Genome Sequencing Psomagen Short- and Long-Read Whole Genome...
Access requires approval by the data custodian. The collection's description and structure are public; querying the data requires an approved request.
Stem Cell Core is published on Target ALS Data Engine.