Modelling human neurodegenerative diseases in research & drug discovery | A new class of scalable, HTS-ready isogenic human cell models

Dr. Mariangela Iovino, Group Leader, Charles River
Dr. Tony Oosterveen, Senior Scientist, bit.bio


Modelling neurodegeneration with ioGlutamatergic Neurons

With a paucity of successful treatments for neurodegenerative diseases, there remains an urgent need to improve research outcomes and drug discovery efficiencies.

It remains challenging to source physiologically-relevant CNS models that are both scalable and reproducible.

Use of human induced Pluripotent Stem Cell (iPSC)-derived neurons offers a relevant model system, but can be hindered by low scalability, heterogeneous populations and long, complex protocols.

A next-generation cell reprogramming technology, opti-ox™, aims to resolve the inconsistencies and inefficiencies of current cell generation methods to enable the precise, consistent and scalable production of human cells. This technology has been used to develop a range of iPSC-derived ioGlutamatergic Neurons carrying disease-specific mutations. The isogenic pairing offers an efficient and robust system using a simple protocol to study neurodegeneration in vitro.

This webcast describes how this new class of precision reprogrammed disease models, are being incorporated into high-throughput screens and drug discovery workflows.

You will learn about the:

  • Need for a new class of scalable isogenic disease models for CNS research and drug discovery
  • Development, characterisation and validation of human cell models with disease-relevant mutations
  • Incorporation of disease ioGlutamatergic Neurons into HTS workflows

Watch on demand now!

Download resource