# A Phenotypical Brain Organoids for Neurodevelopmental Disorders

> **NIH NIH K99** · UNIVERSITY OF CALIFORNIA, SAN DIEGO · 2022 · $105,603

## Abstract

PROJECT SUMMARY/ABSTRACT
Neurodevelopmental disorders (NDDs) are characterized by disrupted development of the brain, and clinically
they lead to impaired neurological function. NDDs account for ~25% of chronic pediatric disease, are incurable
and result in lifelong impairments. Molecular studies have improved our understanding of NDDs, but still large
gaps in knowledge exist. Conventional research tools like the mouse model does not often adequately model
these conditions. Human brain organoids (hBOs) from patient-derived iPSCs can capture many characteristics
and biological events in the developing brain. However, we still do not know how well human NDDs can be
modeled in hBOs. Here, I propose to investigate the range of genotype-phenotype correlations observable in
hBOs from a unique patient derived iPSC library of 750 independent lines to test four hypotheses: 1] NDD-
derived hBOs are both sensitive and specific detectors of the underlying clinical brain pathology. 2] NDD-derived
hBOs show both cellular and molecular hallmarks revealing the stages of disrupted brain development. 3]
Omics approaches applied to these in-vitro derived hBOs can reveal underlying mechanisms of
disrupted development. 4] Gene-environment interactions can be interrogated in hBOs.
The training phase of the award, conducted in Dr. Gleeson’s lab at UCSD, outlines a comprehensive plan for
acquisition of technical and professional skills that will enable my transition to an independent research
position. The successful completion of this project will provide a platform for future experiments aimed to
combine my expertise in stem cell fate decision to gain a deeper understanding of how does these
transcriptional, epigenetic and structural specificities contribute to neurodevelopmental brain disorders.

## Key facts

- **NIH application ID:** 10526025
- **Project number:** 1K99NS125106-01A1
- **Recipient organization:** UNIVERSITY OF CALIFORNIA, SAN DIEGO
- **Principal Investigator:** Lu Wang
- **Activity code:** K99 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $105,603
- **Award type:** 1
- **Project period:** 2022-08-05 → 2024-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10526025

## Citation

> US National Institutes of Health, RePORTER application 10526025, A Phenotypical Brain Organoids for Neurodevelopmental Disorders (1K99NS125106-01A1). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10526025. Licensed CC0.

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