A roadmap for neurodevelopmental disease modeling for non-stem cell biologists

Area of Science:

• Stem cell biology
• Neuroscience
• Translational medicine

Background:

• Induced pluripotent stem cells (iPSCs) are vital for disease modeling but face technical challenges.
• Non-stem cell biologists require guidance for effective iPSC utilization in neurodevelopmental disorder research.

Purpose of the Study:

• To provide an overview of pitfalls and mitigation strategies for using iPSCs in neurodevelopmental disorder modeling.
• To guide experimental planning and execution for researchers less familiar with stem cell disease modeling.

Main Methods:

• The review synthesizes information on experimental design, including appropriate sample sizes.
• It discusses protocols for deriving and purifying human neurons from iPSCs.
• Guidance is provided on the application of gene editing technologies and feasible preclinical study designs.

Main Results:

• The review addresses key questions regarding experimental planning for iPSC-based disease modeling.
• It offers practical advice on sample size, neuron differentiation, gene editing, and preclinical study design.

Conclusions:

• This roadmap aims to empower researchers to establish high-quality human preclinical models for neurodevelopmental disorders.
• Such models facilitate the discovery of disorder-specific phenotypes and advance translational medicine for unmet needs.