Research lines

About Us

Research in OE:

An eye into disease mechanisms. We use purified proteins, engineered cell lines, pluripotent stem cells, and animal models to investigate the pathogenesis of PH, taking advantage of cutting edge computational analysis, sophisticated protein assays, genetic tools, such as the CRISPR/Cas9 technology, and targeted/untargeted multi-omics approaches, to improve diagnosis and to identify molecular targets for potentially new therapeutic approaches.

Looking for new pharmacological therapies. Integrating chemical synthesis and protein studies, we seek to identify potent ligands of enzymes involved in glyoxylate/oxalate metabolism as potential small molecule drugs that comprehensively target all endogenous oxalate sources.

Personalized medicine in PH. In a "one-size-does-not-fit-all" framework, we aim at identifying genetic modifiers and molecular biomarkers to predict disease severity, monitor disease course and anticipate response to therapy, in the perspective of a personalized medicine approach that maximizes the efficacy-to-safety profile.

PH natural history. The consortium maintains a registry of more than one thousand patients whose clinical data are available for research projects aiming at better understanding the natural history of PH, to improve genotype/phenotype correlations and predict disease outcome. Moreover, a collection of data on the response to new RNA interference drugs is ongoing, in order to evaluate long-term effects of these drugs.

The problem of systemic oxalosis. Patients affected by severe PH can undergo renal failure and accumulate oxalate in many tissues, a life-threatening condition called systemic oxalosis. This harmful condition reduces the quality of life of patients, reduces their chances for surgical and non-surgical treatments and complicates their clinical management. Clinical research studies aim at recognizing more and more predictors of systemic oxalosis in order to offer prompt, focused screening of patients.

Contact us if you are interested in getting involved in our research projects