Interplay of Enzyme Therapy and Dietary Management of Murine Homocystinuria.

ABSTRACT: Albeit effective, methionine/protein restriction in the management of classical homocystinuria (HCU) is suboptimal and hard to follow. To address unmet need, we developed an enzyme therapy (OT-58), which effectively corrected disease symptoms in various mouse models of HCU in the absence of methionine restriction. Here we evaluated short- and long-term efficacy of OT-58 on the background of current dietary management of HCU. Methionine restriction resulted in the lowering of total homocysteine (tHcy) by 38-63% directly proportional to a decreased methionine intake (50-12.5% of normal). Supplemental betaine resulted in additional lowering of tHcy. OT-58 successfully competed with betaine and normalized tHcy on the background of reduced methionine intake, while substantially lowering tHcy in

SUBMITTER: Park I 

PROVIDER: S-EPMC7551847 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature