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Experimental gene therapy targets Alzheimer's in High-Risk patients

NCT ID NCT03634007

First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 34 times

Summary

This early-stage study tested a gene therapy called LX1001 in 15 people with a specific genetic form of Alzheimer's (APOE4 homozygotes). The main goal was to check safety and side effects over 52 weeks. Participants had mild cognitive impairment or mild to moderate dementia due to Alzheimer's.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • Duke University

    Durham, North Carolina, 27708, United States

  • K2 Medical Research

    Maitland, Florida, 32751, United States

  • PPD- Orlando Research Unit

    Orlando, Florida, 32806, United States

  • Weill Cornell Medicine

    New York, New York, 10021, United States

Conditions

Explore the condition pages connected to this study.