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NOBEL PRIZE 2026

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NIHNATIONAL INSTITUTE ON AGINGR01

Precision Medicine for Inflammatory Treatment for Alzheimer's Disease in Down Syndrome

Completed
Institution
UNIVERSITY OF SOUTHERN CALIFORNIA, CA
Principal investigator
Robert Rissman
Funding
$604K
Duration
2021–2026
Open portfolio

Moonbase score

Still being scored

Abstract

PROJECT SUMMARY/ABSTRACT Down syndrome (DS) is the most common genetic cause of intellectual disability, with virtually all DS individuals having AD by age 60. Despite exciting advances over the past 20 years in our understanding of DS and how genetics confer susceptibility for AD, there have been few clinical trials to treat AD in the DS population (DS-AD) and currently no means to clinically impact disease progression. We hypothesize that the biological makeup of DS-AD is as heterogeneous as the sporadic AD population and that identifying specific subsets of DS-AD for particular treatments will yield meaningful therapeutic responses. We propose to test a validated biomarker for sporadic AD, our established proinflammatory endophenotype analysis. This analysis will allow for an understanding of differences in immune signaling between DS-AD individuals and how this may impact anti- inflammatory effects on AD biomarkers. For this study we will leverage biobanked samples from the previously completed Phase 3 clinical trial, “Vitamin E in Aged Persons with Down Syndrome (NCT00056329).” For our proposed work we will employ high throughput proteomics on native plasma and exosome subpopulations to validate new techniques and develop a framework for designing better therapeutic trials for DS-AD

Research team

  • Michael S RafiiPI
  • Robert RissmanPI

LOADING MOONBASE SCORE

R01NATIONAL INSTITUTE ON AGINGSpecial Emphasis Panel[ZRG1-BST-W(55)]syndrometherapeutic trialstherapeutic responsesidentifying specificmakeupsporadicanti inflammatorytechniques develop

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