Extracellular Vesicles As Non-Invasive Biomarkers for Huntington Disease Progression and Huntingtin Lowering Therapy
University of Central Florida
Summary
The primary objective of this study is to discover blood-based biomarker of brain Huntingtin (HTT) protein using extracellular vesicles to be used in evaluating target engagement in HTT lowering clinical trials. Secondary objectives of this study include developing more accurate biomarkers of Huntington disease (HD) progression or conversion and to develop standard practices for extracellular vesicle biomarker discovery research. The investigators hypothesize that brain-derived extracellular vesicles (EVs) isolated from human biofluids contain biological cargo specific to their tissue of origin that could allow their use as brain biomarkers for HD. EVs are lipid bilayer-delimited particles that are naturally released from cells in the brain. The investigators will investigate if EVs contents reflect the pathological alterations occurring with disease progression when compared with EVs isolated from biofluids of healthy non-HD persons.
Eligibility
- Age range
- 18–75 years
- Sex
- All
- Healthy volunteers
- Yes
Inclusion Criteria: 1. 18 - 75 years of age 2. can provide informed consent 3. able to read and speak English 4. agree to comply with study procedures (including overnight fasting, blood collection and lumbar puncture); and 5. has been diagnosed with HD (HD Carriers) or not been diagnosed with HD (Non-HD Carriers). Exclusion Criteria: 1. younger than 18 or older than 75 years old 2. known to carry an intermediate CAG repeat between 27 and 39 or a larger expansion of 60 or more CAG repeats 3. receiving nutrition through a tube 4. pregnant 5. participated in a clinical drug trial within 30 da…
Location
- University of Central FloridaOrlando, Florida