HGNC ID: 4296

Target Abstract:

α-Galactosidase A, also known as GLA, is a member of the glycosyl hydrolase 27 family.GLA can be used as a long-term enzyme replacement therapy for patients with established Fabry disease (FD).GLA deficiency triggers FD, a rare X-linked disorder of sphingolipid metabolism, which manifests itself as glycolipid deposition in many tissues.FD is an innate disorder of abnormal catabolism of sphingolipids, and patients with the disease Patients exhibit systemic deposition of ceramide trihexoside (Gb3) and related glycosphingolipids throughout the body in plasma and cellular lysosomes. Characteristic skin lesions (angiokeratomas) in the lower trunk of males are clinically recognized by physicians as FD. In addition, patients may present with ocular deposits, fever, and burning pain in the extremities. Renal failure, cardiac or cerebral complications due to hypertension, or other vascular diseases can lead to death in patients with FD. Deficiency of GLA leads to deposition of glycosphingolipids in blood vessels, which in turn leads to multi-organ disease. In addition to microvascular disease, GLA deficiency can lead to early macrovascular lesions such as stroke and myocardial infarction.

GLA Target Infomation Overview

• Target Symbol: GLA, galactosidase alpha
• Gene Groups: Galactosidases alpha
• Alias: GALA

GLA, galactosidase alpha Target Infomation by Species