Mechanism

HSD17B13

Assets acting on this target.

HSD17B13 (hydroxysteroid 17-beta dehydrogenase 13) is an enzyme produced almost exclusively in the liver, where it sits on the surface of lipid droplets, the intracellular structures that store fat. Its precise biochemical role is still being defined, but human population genetics has been the main driver of interest in this target: certain naturally occurring variants that reduce or eliminate HSD17B13 activity are common in the population and are associated with a lower risk that fatty liver disease will progress to more severe forms involving liver inflammation and scarring (fibrosis), collectively described under the umbrella of metabolic dysfunction-associated steatotic liver disease and its more advanced stage, steatohepatitis. Because people who carry these loss-of-function variants appear generally healthy, the enzyme is considered dispensable, making it an appealing candidate for pharmacological inhibition rather than a target where reduced activity might be expected to cause harm elsewhere in the body. Therapeutic strategies aim to mimic this protective genetic state, either by directly blocking the enzyme's catalytic activity or by reducing production of the protein itself, with the goal of slowing or halting the progression of chronic liver disease.

Research

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