Adenylosuccinic Acid: An Orphan Drug with Untapped Potential

被引：3
作者：
Rybalka, Emma [1 ,2 ,3 ,4 ]
Kourakis, Stephanie [1 ,2 ]
Bonsett, Charles A. [5 ,6 ]
Moghadaszadeh, Behzad [7 ]
Beggs, Alan H. [7 ]
Timpani, Cara A. [1 ,2 ,3 ]
机构：
[1] Victoria Univ, Inst Hlth & Sport IHeS, Melbourne, Vic 8001, Australia
[2] Australian Inst Musculoskeletal Sci AIMSS, Inherited & Acquired Myopathy Program, St Albans, Vic 3021, Australia
[3] Univ Melbourne, Melbourne Med Sch, Dept Med Western Hlth, St Albans, Vic 3021, Australia
[4] Univ Childrens Hosp Basel UKBB, Div Neuropaediat & Dev Med, CH-4056 Basel, Switzerland
[5] Dystrophy Concepts Inc, Indianapolis, IN 46226 USA
[6] Indiana Univ, Sch Med, Indianapolis, IN 46202 USA
[7] Harvard Med Sch, Boston Childrens Hosp, Manton Ctr Orphan Dis Res, Div Genet & Genom, Boston, MA 02115 USA
来源：
PHARMACEUTICALS | 2023年 / 16卷 / 06期
关键词：
adenylosuccinic acid; adenylosuccinate; succinyl-AMP; Duchenne muscular dystrophy; ADSSL1; myopathy; metabolic disease; skeletal muscle; purine metabolism; immunometabolism; Nrf2; activation; PURINE NUCLEOTIDE CYCLE; XANTHINE-OXIDASE; URIC-ACID; OXIDATIVE STRESS; METABOLISM; DYSTROPHIN; FUMARATE; GENE; NRF2; HYPERTROPHY;
D O I：
10.3390/ph16060822
中图分类号：
R914 [药物化学];
学科分类号：
100701 ;
摘要：
Adenylosuccinic acid (ASA) is an orphan drug that was once investigated for clinical application in Duchenne muscular dystrophy (DMD). Endogenous ASA participates in purine recycling and energy homeostasis but might also be crucial for averting inflammation and other forms of cellular stress during intense energy demand and maintaining tissue biomass and glucose disposal. This article documents the known biological functions of ASA and explores its potential application for the treatment of neuromuscular and other chronic diseases.
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页数：20
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