My research focuses on the study of polycystic kidney (PKD) and liver (PLD) diseases; and related disorders. My research has ranged from identification of genes mutated in these diseases and related genes; and the study of expression and function of encoded proteins (particularly as they are excreted in urine subcellular particles known as exosomes) to epidemiology and clinical therapeutic trials.
Current work with my collaborators studies the proteome of exosomes in urine that contain many disease proteins, including the polycystic kidney disease proteins. We have found that the PKD proteins can be readiliy isolated from thes urine particles and are studying how they are altered in disease. We are currently cataloging the proteins in these vesicles in healthy individuals and people with ADPKD.
These observations provide a rationale for diagnostic tests from these diseases and a method to study these disease proteins in people affected with these diseases.
Strategies targeting cyclic nucleotide metabolism in a tissue selective manner thus limiting toxicity. In collaboration with Dr. Larusso's laboratory, we also found that octreotide also reduces reduces liver volumes in people with severe polycystic liver disease and may also decrease kidney growth.
Clinical research and trials towards this goal are funded by:
Analysis of the proteome of PKD-ELVs in polycystic kidney disease and controls American Recovery and Reinvestment Act (ARRA)GRANT 10266762; 1RC1DK086161-01
The PKD Foundation (ADPKD Pain Study)
Industry (Tolvaptan Efficacy and Safety in Management of ADPKD and its Outcomes Phase 2/4, ClinicalTrials.gov, NCT00413777
Tolvaptan Efficacy and Safety in Management of ADPKD and its Outcomes Phase 3/4, ClinicalTrials.gov, NCT00428948
Long-term Efficacy and Safety of Oral Tolvaptan Tablet Regimens in Subjects with ADPKD, ClinicalTrials.gov, NCT01214421
Octreotide for severe Polycystic Liver Disease, ClinicalTrials.gov, NCT00426153