Understanding how a specific gene affects brain development and coordination disorders

Defining the role of Tox3 in congenital cerebellar hypoplasia and ataxia

NIH-funded research Cedars-Sinai Medical Center · NIH-10991047

This study is looking at how a specific gene called TOX3 affects the development of a part of the brain that helps with balance and coordination, and it aims to find out how changes in this gene might be linked to conditions like ataxia and autism, which could help patients understand these disorders better.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionCedars-Sinai Medical Center NIH-funded
Lab location1 site (Los Angeles, United States)
Project IDNIH-10991047 on NIH RePORTER

What this research studies

This research investigates the role of the TOX3 gene in the development of the cerebellum, a brain region crucial for balance and coordination. By studying genetically modified mice, the researchers aim to uncover how changes in this gene can lead to conditions like ataxia and autism. The approach involves examining the genetic and epigenetic factors that influence the formation of cerebellar granule cells, which are essential for normal brain function. Patients may benefit from insights gained about the underlying mechanisms of these disorders.

Who could benefit from this research

Good fit: Ideal candidates for this research are individuals with congenital cerebellar hypoplasia, ataxia, or related developmental disorders.

Not a fit: Patients with conditions unrelated to cerebellar function or those who do not have genetic predispositions linked to the TOX3 gene may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to new understanding and potential treatments for coordination disorders and related conditions.

How similar studies have performed: Previous research has shown promising results in understanding the genetic basis of cerebellar disorders, suggesting that this approach could yield valuable insights.

Where this research is happening

Los Angeles, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.