# Role of ARX mutations in marmoset brain organoids

> **NIH NIH U01** · UNIVERSITY OF TEXAS SAN ANTONIO · 2024 · $20,324

## Abstract

PROJECT SUMMARY/ABSTRACT
Neuropsychiatric disorders represent a leading cause of disability, affecting nearly 19% of the US population.
Only 9% of neuropsychiatric drugs entering clinical trials reach the market, which is one of the lowest success
rates across all therapeutic areas. Fundamental differences between the neurobiology of rodents and humans
have been proposed to account for translational failures in development of effective therapeutic strategies to
mitigate neurological or neurodegenerative diseases or disorders. Rodent behavioral assays are also variably
effective in predicting clinically effective neuropsychiatric drugs. Nonhuman primates (NHPs) are recognized as
a valuable, clinically relevant alternative to span the gap between rodents and humans in the development of
therapies designed to advance brain health. Among NHPs, the common marmoset or Callithrix jacchus (cj)
affords a highly tractable option because of its small size, short lifespan, production of multiple offspring/year
and accurate recapitulation of human neuroanatomy. However, the ultimate utility of the marmoset model
remains in its infancy due to the paucity of efficient tools to facilitate studies requiring genetic modification,
especially those needed to recapitulate complex aspects of brain health. To address this urgent need, we will
combine close proximity to one of two NIH-designated Marmoset Breeding Colonies (U24 MH123422, funded
under RFA-MH-20-145) maintained at the Southwest National Primate Research Center and leading expertise
in brain health and disease in general and the neurogenetics of epilepsy in particular. In this diversity supplement,
we will perform the experiments outlined in Aim 3 of the parent U01 application, which is to assess the impact of
ARX mutations on marmoset cortical neuron development and migration. This aim is designed to advance the
utility of the marmoset model for brain research based on the development of PSC-derived brain organoids and
specific knowledge of the neurological impact of ARX mutations.

## Key facts

- **NIH application ID:** 10946705
- **Project number:** 3U01DA054170-04S1
- **Recipient organization:** UNIVERSITY OF TEXAS SAN ANTONIO
- **Principal Investigator:** Brian Peter Hermann
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $20,324
- **Award type:** 3
- **Project period:** 2021-08-01 → 2026-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10946705

## Citation

> US National Institutes of Health, RePORTER application 10946705, Role of ARX mutations in marmoset brain organoids (3U01DA054170-04S1). Retrieved via AI Analytics 2026-06-12 from https://api.ai-analytics.org/grant/nih/10946705. Licensed CC0.

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