Sirtuin activation as a therapeutic approach against inborn errors of metabolism

J Inherit Metab Dis. 2016 Jul;39(4):565-72. doi: 10.1007/s10545-016-9939-8. Epub 2016 May 4.

Abstract

Protein acylation has emerged as a large family of post translational modifications in which an acyl group can alter the function of a wide variety of proteins, especially in response to metabolic stress. The acylation state is regulated through reversible acylation/deacylation. Acylation occurs enzymatically or non-enzymatically, and responds to acyl-CoA levels. Deacylation on the other hand is controlled through the NAD(+)-dependent sirtuin proteins. In several inborn errors of metabolism (IEMs), accumulation of acyl-CoAs, due to defects in amino acid and fatty acid metabolic pathways, can lead to hyperacylation of proteins. This can have a direct effect on protein function and might play a role in pathophysiology. In this review we describe several mouse and cell models for IEM that display high levels of lysine acylation. Furthermore, we discuss how sirtuins serve as a promising therapeutic target to restore acylation state and could treat IEMs. In this context we examine several pharmacological sirtuin activators, such as resveratrol, NAD(+) precursors and PARP and CD38 inhibitors.

Publication types

  • Review
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acyl Coenzyme A / metabolism
  • Acylation
  • Animals
  • Humans
  • Metabolism, Inborn Errors / therapy*
  • Mice
  • Molecular Targeted Therapy / methods*
  • Protein Processing, Post-Translational*
  • Sirtuins / metabolism*

Substances

  • Acyl Coenzyme A
  • Sirtuins