Understanding how nuclear surveillance is impaired in ALS and FTD

Mechanisms of impaired ESCRT-III nuclear surveillance in ALS/FTD

NIH-funded research Johns Hopkins University · NIH-11125892

This study is looking at how certain problems in brain cells might contribute to ALS and Frontotemporal Dementia, focusing on a specific protein called CHMP7, to help find new ways to treat these conditions.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionJohns Hopkins University NIH-funded
Lab location1 site (Baltimore, United States)
Project IDNIH-11125892 on NIH RePORTER

What this research studies

This research investigates the mechanisms behind the impairment of nuclear surveillance in patients with Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD). By utilizing induced pluripotent stem cell-derived neurons and human brain tissues, the study aims to uncover how disruptions in the nuclear pore complex affect cellular processes critical for neuronal health. The research focuses on the role of a specific protein, CHMP7, in these neurodegenerative diseases, which may lead to better understanding and potential therapeutic targets for ALS and FTD.

Who could benefit from this research

Good fit: Ideal candidates for this research include individuals diagnosed with ALS or FTD, particularly those experiencing early symptoms.

Not a fit: Patients with neurodegenerative diseases unrelated to ALS or FTD may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to new insights and treatments for patients suffering from ALS and FTD.

How similar studies have performed: Previous research has shown promising results in understanding neurodegenerative mechanisms, making this approach both relevant and potentially impactful.

Where this research is happening

Baltimore, 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 age associated neurodegenerative diseaseage associated neurodegenerative disorderage dependent neurodegenerative diseaseage dependent neurodegenerative disorderage-driven neurodegenerative disorders
Last reviewed 2026-06-13 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.