Investigating how to regulate a gene linked to Alzheimer's disease using new delivery methods.

Therapeutic effects of APOE regulation via LNP-RNA formulations in Alzheimer's disease

['FUNDING_R01'] · ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · NIH-11105008

This study is looking at how a specific gene related to Alzheimer's disease affects the risk of memory loss, and it's testing a new way to deliver treatments to the brain that could help improve care for people with this condition.

Quick facts

Phase['FUNDING_R01']
Study typeNih_funding
SexAll
SponsorICAHN SCHOOL OF MEDICINE AT MOUNT SINAI (nih funded)
Locations1 site (NEW YORK, UNITED STATES)
Trial IDNIH-11105008 on ClinicalTrials.gov

What this research studies

This research focuses on Alzheimer's disease (AD), a serious condition that leads to memory loss and cognitive decline. The team is exploring the role of the Apolipoprotein E (ApoE) gene, which has different variants that affect AD risk. They aim to develop lipid nanoparticles (LNPs) that can effectively deliver nucleic acid therapies to the brain, overcoming barriers that typically prevent such treatments from reaching their target. By modifying the expression of ApoE genes, the researchers hope to find new therapeutic strategies to combat Alzheimer's disease.

Who could benefit from this research

Good fit: Ideal candidates for this research include individuals diagnosed with Alzheimer's disease, particularly those with genetic risk factors related to the ApoE gene.

Not a fit: Patients without a diagnosis of Alzheimer's disease or those with other forms of dementia may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to innovative treatments that significantly improve outcomes for patients with Alzheimer's disease.

How similar studies have performed: Previous research has shown promise in using nucleic acid-based therapies for neurodegenerative diseases, but this specific approach targeting ApoE regulation is relatively novel.

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.

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.