How a tiny virus carrier could protect nerve cells in ALS Veterans

Who could benefit from this research

What this research studies

This project uses a novel AAV capsid to deliver SynCav1, a neuroprotective genetic construct, directly to brain and spinal cord motor neurons in ALS models. The goal is to understand how this approach protects neurons at the molecular level and to explore delivery methods that could one day benefit patients, including Veterans with ALS. The work combines animal studies to study delivery, distribution, and functional effects, with a clear aim toward translating findings into human therapies. If successful, this line of research could point toward new treatment strategies to slow disease progression and improve quality of life for those living with ALS.

Why it matters

Where this research is happening

• VA San Diego Healthcare System
  San Diego, United States Active

Researchers

• Principal investigator: Head, Brian P — VA San Diego Healthcare System
• Study coordinator: Head, Brian P

Related research

Other NIH-funded projects on the same topics.

• Investigating how DNA damage and repair mechanisms are altered in ALS and frontotemporal dementia.

  This study is looking at how problems with DNA repair might contribute to conditions like ALS and frontotemporal dementia, and it’s testing ways to fix these issues in cells from patients to help develop new treatments.

  NIH research Methodist Hospital Research Institute · Houston, United States · NIH-9980670

• Understanding how work exposures and genes influence ALS risk

  A study is building a long-term group of people at higher risk for ALS to see how their jobs, exposures at work, and genes together affect if and when the disease might develop.

  NIH research University of Michigan at Ann Arbor · Ann Arbor, United States · NIH-11631673

• NUZ-001: a treatment option for ALS to protect nerve cells

  A small molecule called NUZ-001 is being tested to help people with ALS by improving cell systems that keep proteins balanced and prevent harmful protein clumps.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-11564156

• Alpha5 integrin's role in ALS: how a cell switch worsens disease

  Aims to understand how a specific protein called alpha5 integrin in immune cells affects ALS progression and whether blocking it can help extend life.

  NIH research Cedars-Sinai Medical Center · Los Angeles, United States · NIH-11540166

• C9orf72 ALS/FTD Prevent: prepping for future treatment

  This grant aims to get ready for a preventive treatment for people who carry the C9orf72 genetic change that can lead to ALS or frontotemporal dementia.

  NIH research University of Miami School of Medicine · Coral Gables, United States · NIH-11431628

• Air pollutants and ALS: mapping environmental links to motor neuron disease

  This NIH grant explores whether different air pollutants are linked to the development of ALS and uses a large U.S. data set to look for environmental risk factors.

  NIH research Washington University · Saint Louis, United States · NIH-11423391

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.