Fabry Disease Models

Autoimmune Disease Models
Autoimmune Nephropathy ModelsAsthma ModelsSystemic Lupus Erythematosus (SLE) ModelsSjögren's Syndrome ModelsAutoimmune Dermatoses Models Neuroimmune Disease ModelsInflammatory Bowel Disease (IBD) ModelsRheumatoid Arthritis ModelsImmune Reconstitution Autoimmune Disease Models
Metabolic Disease Mouse Models
Obesity ModelsDiabetes ModelsAndrogenetic Alopecia (AGA) ModelsMASH ModelsChronic Liver Fibrosis ModelsAcute Liver Injury ModelsChronic Kindey Disease (CKD) ModelsCholestasis ModelsAlcohol-related Liver Disease (ALD) ModelsActue Kindey Disease (AKD) ModelsDiabetes Nephropathy (Diabetic Kidney Disease) ModelsOsteoporosis ModelsSarcopenia ModelsGout/Hyperuricemia ModelsFemale Reproduction Models
Wild Mouse 750
Wild Mouse 750
Rare Disease Mouse Models
Hemophilia ModelsProgressive Familial Intrahepatic Cholestasis ModelsAutosomal Dominant Polycystic Kidney Disease (ADPKD)Glycogen Storage Disease type 1a ModelGrowth Failure ModelsFabry Disease ModelsHepatolenticular Degeneration ModelNiemann-Pick Disease (Sphingomyelinosis) ModelsHypophosphatasia ModelGM2 Gangliosidoses ModelPulmonary Alveolar Proteinosis ModelLimb Girdle Muscular Dystrophies ModelMaple Syrup Urine Disease ModelMucopolysaccharidosis ModelsHutchinson-Gilford Progeria Syndrome ModelPhenylketonuria/Hyperphenylalaninemia ModelsUrea Cycle Disorders ModelsTyrosinemia ModelThalassemia Models
Neurological Disease Mouse Models
Alzheimer's Disease (AD) ModelsHumanized Alzheimer's Disease (AD) ModelsParkinson's Disease (PD) ModelsHuntington's Disease (HD) ModelsAmyotrophic Lateral Sclerosis (ALS) ModelsTransthyretin Amyloidosis (ATTR) ModelsPain Mouse ModelsDuchenne Muscular Dystrophy (DMD) Models
Oncology Mouse Models
Human Xenograft ModelsDrug ResistanceT-Cell EngagersHumanized Immune Checkpoint ModelsHumanized Immune System ModelsSpontaneous Tumor Models
Cardiovascular Disease Mouse Models
Heart Failure ModelsHypertension ModelsAtherosclerosis ModelsAdvanced Cardiovascular and Metabolic Complication ModelsHyperlipidemia ModelsHypertropic Cardiomyopathy ModelsStroke ModelsThrombosis Models
Humanized Immune System Models
huPBMC-NCGhuHSC-NCG-XhuHSC-NCG-hIL15huHSC-NCG
Organs or Tissue Humanized Mouse Models
Wild Mouse 765
Wild Mouse 765

Fabry disease (FD) is an X-linked lysosomal storage disorder caused by deficient or absent α-galactosidase A (α-GAL) activity, leading to impaired glycosphingolipid metabolism. With an estimated prevalence of 1:40,000 to 1:117,000 in the general population, the pathogenesis of this disease involves the progressive accumulation of globotriaosylceramide (GL-3) and its deacylated form, lyso-GL-3 (globotriaosylsphingosine), due to the enzymatic deficiency. Key target organs include the cardiovascular system, renal parenchyma, and cerebrovascular network.

 

● Genetically Engineered FD Mouse Models

Strain No.
 Strain Name Strain Type Description
T057359 NCG-Gla-KO Knockout GLA enzyme activity is significantly reduced in the plasma, brain, kidney, liver and heart of NCG-Gla-KO male hemizygous mice. Increased deposition of Gb3 and Lyso-Gb3 is also detected in these tissues. Immunodeficiency of this model allows the evaluation of cell-based therapies against Fabry disease
T012717 Gla-KO Knockout GLA enzyme activity is significantly reduced in the plasma, brain, kidney, liver and heart of Gla-KO male hemizygous mice. Increased deposition of Gb3 and Lyso-Gb3 is also detected in these tissues, indicating Fabry disease like phenotypes