Exploring how gene mutations in cilia affect important proteins for cell growth and development

Understanding how ciliary gene mutations affect the processing and activity of Gli2 and Gli3 transcription factors

NIH-funded research Weill Medical Coll of Cornell Univ · NIH-10833102

This study is looking at how changes in certain genes related to tiny hair-like structures in our cells can affect important proteins that help control cell growth and development, which could lead to new insights and treatments for people with ciliopathies.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionWeill Medical Coll of Cornell Univ NIH-funded
Lab location1 site (New York, United States)
Project IDNIH-10833102 on NIH RePORTER

What this research studies

This research investigates the role of ciliary gene mutations in the processing and activity of Gli2 and Gli3 transcription factors, which are crucial for cell growth and differentiation. By examining how these mutations impact the signaling pathways that regulate embryonic development and various cellular functions, the study aims to uncover the underlying mechanisms of ciliopathies, a group of disorders caused by ciliary dysfunction. The research employs advanced molecular biology techniques to analyze the phosphorylation and degradation processes of these transcription factors in response to signaling molecules. Patients may benefit from a better understanding of their conditions and potential new therapeutic targets.

Who could benefit from this research

Good fit: Ideal candidates for this research include individuals with congenital abnormalities or ciliopathies that may be related to ciliary gene mutations.

Not a fit: Patients without any known genetic mutations affecting ciliary function or those with unrelated conditions may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to improved treatments for patients with developmental abnormalities linked to ciliary dysfunction.

How similar studies have performed: Previous research has shown promising results in understanding the role of cilia in various diseases, indicating that this approach could yield valuable insights.

Where this research is happening

New York, 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.
Conditions Cancersneoplasm/cancer
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.