Targeting brain cells to improve treatment for Alzheimer's disease

Targeting brain myeloid cells with siRNA-lipid conjugates for treatment of Alzheimer's disease

['FUNDING_R01'] · VANDERBILT UNIVERSITY · NIH-11073756

This study is testing a new way to deliver a special treatment to brain cells that could help reduce the harmful effects of Alzheimer's disease, making it easier for the body to clear out a substance that contributes to the condition.

Quick facts

Phase['FUNDING_R01']
Study typeNih_funding
SexAll
SponsorVANDERBILT UNIVERSITY (nih funded)
Locations1 site (Nashville, UNITED STATES)
Trial IDNIH-11073756 on ClinicalTrials.gov

What this research studies

This research focuses on developing a new method to deliver small interfering RNA (siRNA) to brain myeloid cells, which play a crucial role in Alzheimer's disease. By using a novel lipid conjugate, the study aims to enhance the delivery and effectiveness of siRNA that can reduce the expression of genes associated with Alzheimer's, such as CD33. The approach involves administering the siRNA into the cerebrospinal fluid, where it can be transported effectively to target cells throughout the brain. This innovative method seeks to improve the clearance of amyloid beta, a key factor in Alzheimer's pathology.

Who could benefit from this research

Good fit: Ideal candidates for this research are individuals diagnosed with Alzheimer's disease or those at risk for developing it due to genetic factors.

Not a fit: Patients with other forms of dementia or neurological disorders unrelated to Alzheimer's may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to more effective treatments for Alzheimer's disease by improving the clearance of harmful proteins in the brain.

How similar studies have performed: Previous research has shown promise in using similar oligonucleotide approaches for targeting brain cells, indicating potential for success in this novel application.

Where this research is happening

Nashville, 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.

View on NIH RePORTER →

Last reviewed 2026-05-15 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.