First-of-its-Kind gene therapy injected into spine to fight Alzheimer's

NCT ID NCT03634007

Summary

This early-stage study tested the safety of a new gene therapy, LX1001, for people with Alzheimer's disease who carry a specific genetic risk factor (two copies of the APOE4 gene). The therapy was injected into the spinal fluid of 15 participants over a year to see if it was safe and to find the right dose. The goal was to see if the treatment could change a key protein in the brain linked to Alzheimer's progression.

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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.