Researchers
Rafii, Michael S MD
University of Southern California
INCLUDE Grants
Clinical trials to prevent Alzheimer’s Disease in Down Syndrome
Grant Number
3R61AG066543-02S1
NIH Institute
NIA
Mechanism
R61
AD remains one of our greatest unmet medical needs, without any approved disease-modifying therapies. Individuals with DS comprise the largest group with genetically determined AD. We propose to assess the utility of Tau PET imaging as well as CSF and blood levels of Ptau217 as outcome measures for clinical trials of AD in adults with DS.
Precision Medicine for Inflammatory Treatment for Alzheimer’s Disease in Down Syndrome
Grant Number
R01AG073979
NIH Institute
NIA
Mechanism
R01
Down Syndrome (DS) is an Alzheimer’s disease related disorder (ADRD) that involves traditional AD neuropathology and cognitive impairment at an accelerated rate. We propose to use well- characterized, biobanked plasma samples from the Vitamin E in DS trial to characterize AD biomarkers and develop a precision medicine approach for use of anti-inflammatories in DS trials. In this proposal we will utilize our expertise in high throughput proteomics and analytics to better characterize our sample set and expand the database for this trial.
Publications
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Alzheimer's & dementia : the journal of the Alzheimer's Association
Current advances and unmet needs in Alzheimer’s disease trials for individuals with Down syndrome: Navigating new therapeutic frontiers. -
Alzheimer's & dementia : the journal of the Alzheimer's Association
The history of Down syndrome-associated Alzheimer’s disease; past, present, and future. -
Neurobiology of aging
Differences in cholinergic terminal density in adults with Down syndrome compared to neurotypical controls measured by [18F]-fluoroethoxybenzovesamicol positron emission tomography imaging. -
Alzheimer's & dementia : the journal of the Alzheimer's Association
Brain cholinergic terminal density utilizing [18F]-fluoroethoxybenzovesamicol PET in adults with Down’s syndrome: Relationship to amyloid PET and cognitive performance. -
Alzheimer's & dementia : the journal of the Alzheimer's Association
The mediating role of plasma glial fibrillary acidic protein in amyloid and tau pathology in Down’s syndrome. -
Journal of neurology
De novo variants in immune regulatory genes in Down syndrome regression disorder. -
Alzheimer's & dementia : the journal of the Alzheimer's Association
Assessing amyloid PET positivity and cognitive function in Down syndrome to guide clinical trials targeting amyloid. -
Lancet (London, England)
Addressing challenges in health care and research for people with Down syndrome. -
American journal of medical genetics. Part A
Safety and tolerability of intravenous immunoglobulin infusion in Down syndrome regression disorder. -
The Lancet. Neurology
Comparison of tau spread in people with Down syndrome versus autosomal-dominant Alzheimer’s disease: a cross-sectional study. -
JAMA neurology
Safety, Tolerability, and Immunogenicity of the ACI-24 Vaccine in Adults With Down Syndrome: A Phase 1b Randomized Clinical Trial.