Development of recombinant Lecithin Cholesterol Acyltransferase as a therapeutic

National Heart, Lung, And Blood Institute

Investigators

Linked publications, trials & patents

Abstract

In the past year, we have mostly focused on developing animal models to investigate the effect of recombinant LCAT(rLCAT) infusion therapy on renal function and to identify possible other indications for the the therapy. We have completed and published a study showing that rLCAT treatment can prevent proteinuria and renal dysfunction in a mouse model of LCAT deficiency, which supports our future plans for a clinical trial. We are developed a drug induced cholestasis model for LpX formation, the main abnormal lipoprotein nephrotoxic particle that accumulates in the absence of LCAT. We showed that rLCAT may also be useful for lowering LpX in cholestasis, which is much more common than the genetic deficiency of LCAT. We also published two methodological papers on new assays for measuring LCAT activity and LpX, which we plan to use in our future clinical trials.