Data catalog

Target ALS

Target ALS

Accelerate your Amyotrophic Lateral Sclerosis (ALS) research with the Target ALS Data Collection – a comprehensive ecosystem of integrated, critical resources provided with minimal restrictions. Overcome common research barriers by accessing standardized, well-characterized components designed to fuel discovery:  

  • Natural History Study (Longitudinal Biofluids Core): Leverage unique longitudinal biofluids (CSF, blood, urine) paired with deep clinical, genomic, and functional data collected from a diverse, actively enrolling cohort of ALS participants and healthy controls.
  • Postmortem Tissue Core: Explore human Central Nervous System (CNS) tissue linked to rich clinical, histological, and multi-omic data for direct investigation of human pathology.
  • Stem Cell Core: Utilize patient-derived induced Pluripotent Stem Cell (iPSC) lines (representing familial, sporadic, and control cases, including isogenic lines) for disease modeling and therapeutic screening.
     

Integrate insights across these unique resources – tissue, cells, and longitudinal fluid/clinical data – to investigate ALS mechanisms, discover and validate novel biomarkers, and drive the development of effective treatments.

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Partner datasets
Target ALS
Amyotrophic Lateral Sclerosis (ALS)
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Datasets

Target ALS

Data Collection Time Frame

2016-Present

Participants

225*

Geographical Coverage

International

Usage Examples

  • Investigating ALS pathophysiology using human postmortem tissue, patient-derived cell models (iPSCs), and longitudinal biofluid analysis

  • Discovering and validating diagnostic, prognostic, progression, and pharmacodynamic biomarkers in biofluids (CSF, blood, urine)

  • Identifying and validating novel therapeutic targets using multi-omics data across different biological sample types

  • Modeling disease mechanisms and testing therapeutic efficacy (including gene therapies) in human iPSC-derived cells

  • Understanding ALS heterogeneity and progression patterns by integrating clinical, functional, genetic, and molecular data over time across diverse populations

  • Correlating clinical phenotypes with neuropathology or molecular signatures.

Data Modalities

Genomics

  • Whole Genome Sequencing (WGS - Short and Long Read)
  • Gene Mutation Status

Transcriptomics

  • Bulk RNA Sequencing (RNA Seq)
  • Spatial Transcriptomics

Proteomics

  • Targeted Proteomics
  • Untargeted Proteomics
  • Protein Biomarker Assays

Clinical Data

  • Clinical Assessments / Scales
  • Demographics
  • Medical History
  • Clinical Laboratory Tests

Biosamples

  • Blood (incl. Plasma, Serum, PBMCs)
  • Cerebrospinal Fluid (CSF)
  • Urine
  • Postmortem Tissue

Derived Cellular Models

  • iPSC Lines (Patient, Control, Isogenic)

Pathology

  • Quantitative Pathology Data

Functional Measures

  • Digital Biomarkers

Biomarkers

  • Fluid Biomarker Levels (e.g., NfL)

Other / Contextual

  • Environmental and Occupational History Questionnaire

Interested in accessing this data?
 

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