What We Do
We combine our capabilities with those of our partners to:
- Use induced pluripotent stem cells (iPSCs) and iPSC-derived neurons to study the molecular basis of neurological diseases
- Develop new tools and technology in disease modelling and target engagement that will help to identify potential therapies
- Translate newly developed assays onto an automated high-throughput platform that can be integrated with preclinical drug screens
C-BIGxEDDU
Watch our video with Nicolas Ferry, from the Clinical Biospecimen Imaging and Genetic Repository (C-BIG), and Taylor Goldsmith from The Neuro's EDDU (Early Drug Discovery Unit) and learn how we are collaborating to accelerate the discovery of new treatments for neurological disorders benefiting patients worldwide.
Discover Our Working Groups
- iPSC phenotyping and CRISPR editing
- Disease Mechanisms and Discovery Assays
- Brain Organoids and 3D Cultures
- Training and outreach
iPSC phenotyping and CRISPR editing
iPSCs are at the heart of research at the EDDU.
This working group is responsible for characterizing iPSC lines that come through the EDDU to ensure that only high-quality iPSCs are used in our research projects. The group is further expanding their library of iPSC lines by generating gene-edited versions using CRISPR technology.
iPSC phenotyping
- Optimization of growth media and passaging reagents
- Quality control testing including chromosomal integrity, morphological features, pluripotency marker expression, microbiological/viral screening
CRISPR editing
- Genetic knockin or knockout (KO) in control lines
- Correction of disease-associated mutations in patient lines
Antibody Validation and CRISPR KO
- Generation of genetic knockout lines to validate commercial antibodies (ALS Reproducible Antibody Platform [ALS-RAP])- partnership with an antibody validation company YCharOS
Disease Mechanisms and Discovery Assays
Automation is driving research forward at the EDDU.
With state-of-the-art equipment, this group is working to bring our assays with iPSC-derived cells into screenable 2D and 3D formats.
"In-house" tools for image acquisition and analysis
- Optimization of immunofluorescence-based assays
- Established workflow for data management
- Open-source software developed in house to enhance data analysis
Focus on multiple disease areas:
- Parkinson’s disease and other synucleinopathies
- Neuromuscular diseases
- Neurodevelopmental diseases
- Neuroimmune and Neuroinflammation
Brain Organoids and 3D Cultures
3D neuronal organoids are shaking up our perspective on the human brain.
Taking iPSC-derived neurons beyond two dimensions has allowed this group to establish the EDDU’s organoid program. The team is applying multiple approaches to characterize 3D neuronal organoid models, while also exploring innovative 3D culturing systems.
3D model development
- Multiple tissue types: forebrain, midbrain, cerebral
- Customizable batch numbers (100 to 700) and age (15 days to 100+ days)
Histology and 3D imaging
- Immunohistochemistry and immunofluorescence microscopy
- Parafilm embedding and microtome sections
- Cubic tissue clearing
- Methods for automated 3D imaging on Opera Phenix HCs
Organoid maintenance and quality control
- 15K minibrain capacity in organoid-specific incubators
- Dedicated team oversees day-to-day monitoring of each individual organoid using a Laboratory Information Monitoring System (LIMS)
Single cell phenotyping
- Comprehensive analysis of individual cell types in organoids using our state-of-the-art, automated Attune Acoustic Flow Analyzer
- Workflow for isolation of single cells from organoids for flow analysis or single-cell sequencing analysis
- Informatics pipeline for handling single-cell expression data of specific cell types within organoids
Training and outreach
Train and learn with us at the EDDU.
For more information about our workshops, seminars, software and protocols, and other training resources, please visit our Training and Outreach page.
