CRISPR Applications in Metabolic Disease Treatment: A Case of Hyperuricemia

Abstract

The condition of hyperuricemia, which refers to elevated uric acid levels in blood, has become a widespread metabolic disorder that leads to gout development and increases the risk of various metabolic and cardiovascular and renal diseases. The number of people with this condition continues to rise across the globe. The formation of monosodium urate crystals in joints leads to gout, which causes severe joint inflammation that severely impacts the life quality of patients. The current treatment options for hyperuricemia include allopurinol and uricosurics which either reduce uric acid production or enhance its elimination but patients need to continue their medication forever. The treatment fails to resolve the underlying condition. The rapid development of CRISPR/Cas9 gene editing technology during the last few years created a new method to address the fundamental causes of metabolic diseases including hyperuricemia. The paper examines the potential of CRISPR technology to treat metabolic diseases through the example of hyperuricemia treatment. The research investigates multiple approaches which work to achieve uric acid homeostasis and optimize urate transport and regulating uric acid production related metabolic pathways. The research examines current gene editing systems and new delivery techniques which enable successful in vivo applications. The paper presents current developments while identifying remaining obstacles that prevent laboratory breakthroughs from reaching clinical practice and suggests future directions for CRISPR hyperuricemia treatment.