Developing a drug screening platform for neurological diseases

About the project:

Neurodevelopmental disorders are a large group of conditions resulting from damage occurring during critical periods of brain development, while growing in the womb or during the first years of life.

Increased brain size, also known as megalencephaly, is often reported in patients with neurodevelopmental conditions such as autism spectrum disorder (ASD), intellectual disability (ID), attention deficit hyperactivity disorder (ADHD), and epilepsy.

Currently, there is no cure for most neurodevelopmental disorders and the only treatments available for children are directed at alleviating their symptoms. Advancements in stem cell research, in the form of 3D brain organoids, now offer a unique opportunity to study neurodevelopmental disorders “in a dish” and to develop effective and targeted treatments for these conditions.

In this project, we will use cutting edge brain organoid technology to develop a high-throughput drug screening platform for Sotos syndrome, a common overgrowth condition with megalencephaly and ID, affecting 1 in 10,000 to 1 in 14,000 individuals.

Research theme: reSOLVE

Tissue of interest: Brain

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The Novo Nordisk Foundation Center for Stem Cell Medicine, reNEW, is supported by a Novo Nordisk Foundation grant number NNF21CC0073729​​