Disease

Gaucher Disease

About the Disease
Gaucher's Disease, also known as gaucher disease, is related to gaucher disease, type ii and gaucher disease, type iii, and has symptoms including apnea, muscle rigidity and muscle spasticity. An important gene associated with Gaucher's Disease is GBA1 (Glucosylceramidase Beta 1), and among its related pathways/superpathways are Metabolism and Sphingolipid metabolism. The drugs Sofosbuvir and Ledipasvir have been mentioned in the context of this disorder. Affiliated tissues include spleen, liver and bone marrow, and related phenotypes are splenomegaly and hepatomegaly

Common Targets
CPVL | Histone deacetylase (nonspecified subtype) | Glucosidase (nonspecified subtype) | LYZ | GBA1 | MPHOSPH8 | Glycogen phosphorylase (nonspecified subtype) | LRRK2 | ASAH1 | UGCG | CHIT1 | Chaperone (nonspecified subtype) | CMYA5 | HLA-B | SLC5A4 | GUSB | PSAP | ZNF737 | HLA-DRB1 | HMMR | HLA-A | USH2A | ERN1 | Heat shock protein 70 (nonspecified subtype) | CACNA1A | PCDHB8 | GBA2 | GLB1 | MEST | DNAJC5 | GAA | NPC1 | GALC | TEAD3 | alpha-Mannosidase (nonspecified subtype) | GLA

疾病靶点研报
Gaucher Disease

Note: If you'd like to get a target analysis report for Gaucher Disease, or if you are interested to learn how our AI-powered BDE-Chem can design therapeutic molecules to interact with the target(s) above against the disease of Gaucher Disease at a cost 90% lower than traditional approaches, please feel free to contact us at BD@silexon.ai.

Other Diseases

Atopic Dermatitis | Hypothalamic Obesity | Bleeding Disorder Due To CalDAG-GEFI Deficiency | Prurigo Nodularis | Distal Myopathy | Neurocutaneous Melanocytosis | Familial Mediterranean Fever | Antisocial Personality Disorder | Schistosomiasis Mansoni | Hereditary Spastic Paraplegia | Hamartoma | Delirium | Zollinger-Ellison Syndrome | Cannabis Abuse | Progressive Encephalopathy-optic Atrophy Syndrome | Hyperthermia, Malignant | Porphyria, Acute Intermittent | Eiken Syndrome | Hyperlipidemia, Familial Combined | AIDS | Spondyloperipheral Dysplasia | Arthritis, Reactive | Nestor-Guillermo Progeria Syndrome | Pancreatitis | Unverricht-Lundborg Syndrome | Dupuytren Disease | Congenital Stationary Night Blindness | Proctitis | Methylmalonic Aciduria And Homocystinuria, CblC Type | Lymphedema | Macrophage Activation Syndrome | CEDNIK Syndrome | Craniopharyngioma | Epidermolysis Bullosa Simplex | Poikiloderma With Neutropenia | Cleidocranial Dysplasia | Thromboembolism | Pineoblastoma | Diabetes Type 1 | Primary Biliary Cholangitis | Myocarditis | Proopiomelanocortin Deficiency | Vitelliform Macular Dystrophy | Majeed Syndrome | Jalili Syndrome | Optic Atrophy 2 | C3 Glomerulonephritis | Endometritis | Acrodermatitis | Osteopathia Striata With Cranial Sclerosis | Congenital Myasthenic Syndrome | Primary Aldosteronism | Discoid Lupus Erythematosus | Hereditary Hemorrhagic Telangiectasia Type 2 | Meier-Gorlin Syndrome | Tularemia | Scabies | Rotor Syndrome | Schizophrenia | Placenta Previa | B-cell Prolymphocytic Leukemia | Parapsoriasis | Paraganglioma, Carotid Body | Autonomic Neuropathy | Angioedema, Hereditary | Primary Cutaneous Amyloidosis | Microcephaly | Familial Hyperaldosteronism | Neurofibromatosis Type 2 | Rhinitis | Diffuse Palmoplantar Keratoderma | Citrullinemia | Reye Syndrome | Rheumatoid Arthritis | Dystonia | Hydronephrosis | PHARC Syndrome | C3 Glomerulopathy | Neovascular Glaucoma | Zimmermann-Laband Syndrome | Scleroderma | Ataxia-ocular Apraxia 2 | Glioblastoma | Essential Fructosuria | Persistent Truncus Arteriosus | Anovulation | Transthyretin-related Amyloidosis | Retinopathy Of Prematurity | Hypoalbuminemia | Benign Recurrent Intrahepatic Cholestasis 1 | Facioscapulohumeral Muscular Dystrophy | Multiple Myeloma | Hashimoto Thyroiditis | Platelet Disorders | Richter's Syndrome | Polymyositis | Dysgerminoma | Spinocerebellar Ataxia Type 14 | Glycogen Storage Disease Type 1a | Hemolytic Uremic Syndrome, Atypical | Autosomal Recessive Congenital Ichthyosis | Snyder-Robinson Syndrome | Chromosome 8q21.11 Deletion Syndrome | Gardner Syndrome | Osteoporosis | Pelizaeus-Merzbacher Disease | Leukemia | Retinitis Pigmentosa 3 | Erythema Multiforme | Sclerosteosis | Centronuclear Myopathy | Specific Granule Deficiency | Camurati-Engelmann Disease | Anorchia | Metabolic Syndrome | Encephalitis, Tick-borne | Cryptorchidism | Leiomyoma | Pemphigus Vulgaris | Aneurysm, Thoracic Aortic | Giant Axonal Neuropathy | Incontinentia Pigmenti | Transient Bullous Dermolysis Of The Newborn | Myopia | Polymyalgia Rheumatica | Overactive Bladder | Trichothiodystrophy | Keratosis, Actinic | Primary Hyperoxaluria | Autosomal Recessive Spastic Paraplegia Type 75 | Pleural Tuberculosis | Posterior Polar Cataract | Spinal And Bulbar Muscular Atrophy | Giant Cell Arteritis | Periventricular Nodular Heterotopia | Schnitzler Syndrome | Hodgkin Lymphoma | Coffin-Siris Syndrome | Spinocerebellar Ataxia Type 31 | Sepiapterin Reductase Deficiency | LRBA Deficiency | Cardiospondylocarpofacial Syndrome | Hypodontia | Pre-eclampsia | Sleep Apnea, Central | Inflammatory Myofibroblastic Tumor | DiGeorge Syndrome | Otopalatodigital Syndrome Type 2 | Hyperoxaluria | Craniolenticulosutural Dysplasia | Shock, Cardiogenic | Skin Papilloma | Albinism | Mast Cell Leukemia | Hereditary Xerocytosis | Uveitis, Anterior | Palmoplantar Keratoderma | Nicolaides-Baraitser Syndrome | Prolactinoma | Multiple Epiphyseal Dysplasia | Varicocele | Myocardial Infarction | Familial Retinal Arterial Macroaneurysm | Colorectal Adenoma | Bronchiolitis | Dyggve-Melchior-Clausen Disease | Metabolic Diseases | Tumoral Calcinosis | Liver Failure, Acute Infantile | Alpha-thalassemia Myelodysplasia Syndrome | Hemoglobinopathies | Gangliosidosis, GM1 | Deafness, Dystonia, And Cerebral Hypomyelination | Endometrial Hyperplasia | Pyoderma Gangrenosum | Ichthyosis Bullosa Of Siemens | Long-chain 3-hydroxyacyl-coenzyme A Dehydrogenase Deficiency | Lesch-Nyhan Syndrome | Neurocutaneous Syndromes | Glanzmann Thrombasthenia | Neuroectodermal Tumors, Primitive | Chronic Idiopathic Myelofibrosis | Atrial Septal Defect | Ectodermal Dysplasia | Chronic Beryllium Disease | Peritonitis | POEMS Syndrome | Hyperglycemia | Dengue Hemorrhagic Fever | Spinocerebellar Ataxia Type 6 | Osteogenesis Imperfecta Type I | Hereditary Elliptocytosis | Osteitis | Cheilitis | 3-methylglutaconic Aciduria Type IV | Hereditary Sensory Neuropathy Type 1 | Hypolipoproteinemia | Nemaline Myopathy 8 | Chronic Granulomatous Disease | Acral Lentiginous Melanoma | Mitochondrial DNA Depletion Syndrome, Encephalomyopathic Form | Auriculocondylar Syndrome | Shwachman-Bodian-Diamond Syndrome | Nevus | Acute Coronary Syndrome | Chondroma | Hypermetropia | Kernicterus | Chromosome 5q Deletion Syndrome | Peeling Skin Syndrome, Acral Type | Methemoglobinemia Type IV | Goiter, Nodular | Primary Carnitine Deficiency | Histiocytic Sarcoma | Sarcomatoid Carcinoma Of The Lung | Hypohidrotic Ectodermal Dysplasia | Methemoglobinemia | Aspergillosis | Extramammary Paget's Disease | Erythematotelangiectatic Rosacea | Cholangiocarcinoma | Pontocerebellar Hypoplasia | Mohr-Tranebjaerg Syndrome | IMAGe Syndrome | Aplastic Anemia | Gastritis, Atrophic | Otosclerosis | Bipolar Disorder | Retinal Degeneration | Tuberculosis | Pyruvate Carboxylase Deficiency Disease | Melnick-Needles Syndrome | Mitochondrial Disease | Ganglioneuroma | Microtia | Beare-Stevenson Syndrome | Exostoses | Spondylocostal Dysostosis | Schnyder Crystalline Corneal Dystrophy | Hypertensive Nephropathy | MELAS Syndrome | Spastic Paraplegia Type 7 | Alpha-mannosidosis | Pernicious Anemia | ADNP Syndrome | Thin Basement Membrane Disease | Intermittent Claudication | Ureteropelvic Junction Obstruction | Dent Disease | Subacute Sclerosing Panencephalitis | Rhabdoid Tumor | Meningitis | Demyelinating Diseases | Cystitis | Atelosteogenesis Type 1 | T-cell Leukemia | Thrombocythemia, Essential | Cornelia De Lange Syndrome | Diabetes Gestational | Disseminated Superficial Actinic Porokeratosis | 3-methylcrotonyl-CoA Carboxylase Deficiency | Eosinophilic Asthma | Multiple Congenital Anomalies-hypotonia-seizures Syndrome 2 | Epidermolysis Bullosa Simplex With Mottled Pigmentation | Chloridorrhea, Congenital | Pancytopenia | Epidermal Nevus Syndrome | Mitochondrial Cytopathy | Melanocytic Nevus | Adenylosuccinate Lyase Deficiency | Cryoglobulinemia | Chediak-Higashi Syndrome | 3-hydroxy-3-methylglutaric Aciduria | Malignant Fibrous Histiocytoma | Liddle Syndrome | Pain | Neuroma | Cockayne Syndrome | Familial Thoracic Aortic Aneurysm | Maple Syrup Urine Disease | Stromal Corneal Dystrophy | Esophageal Adenocarcinoma | Pseudoachondroplasia | Trismus-pseudocamptodactyly Syndrome | Epidermolysis Bullosa | Hepatitis A | Mitochondrial DNA Depletion Syndrome 13 | Pigment Dispersion Syndrome | Amyloidosis | Lyme Disease | Renal Hypomagnesemia 3 | Chronic Inflammatory Demyelinating Polyneuropathy | X-linked Charcot-Marie-Tooth Disease | Measles | Purpura | Azoospermia | Prostatitis | Macular Degeneration | Hypobetalipoproteinemias | Sarcoidosis | Bursitis | Torticollis | Ehlers-Danlos Syndrome With Progressive Kyphoscoliosis, Myopathy, And Hearing Loss | Ectopia Lentis, Isolated, Autosomal Recessive | Angiodysplasia | Postpoliomyelitis Syndrome | Joubert Syndrome 2 | Silicosis | Cutaneous T-cell Lymphoma | Tylosis With Esophageal Cancer | Sialidosis | Ulcerative Colitis | Nager Acrofacial Dysostosis | HELLP Syndrome | Aneurysm, Abdominal Aortic | Fetal And Neonatal Alloimmune Thrombocytopenia | Hypogonadism | Paronychia | Light Chain Amyloidosis | Corticobasal Syndrome | Schizotypal Personality Disorder | Cerebrovascular Disorders | Cystinuria | Chudley-McCullough Syndrome | Benign Familial Neonatal Convulsions | Glycogen Storage Disease Type 1b | Intestinal Hypomagnesemia 1 | Hemimegalencephaly | Hemorrhoids | Recurrent Respiratory Papillomatosis | Osteonecrosis Of The Jaw | Spinal Muscular Atrophy | Acute Leukemia | Acute Lymphocytic Leukemia | Primary Erythromelalgia | Galloway-Mowat Syndrome | Muckle-Wells Syndrome | Schizencephaly | Glomerulonephritis | Carbonic Anhydrase VA Deficiency | Porphyria | Mitochondrial Encephalomyopathy | Asperger Syndrome | Platyspondylic Lethal Skeletal Dysplasia, Torrance Type | Sarcoma | Lymphoma, Follicular | Obesity, Morbid | Thymoma, Malignant | Spinocerebellar Ataxia Type 20 | Stevens-Johnson Syndrome | Arthritis, Psoriatic | Sleep Apnea | Microcephalic Osteodysplastic Primordial Dwarfism, Type II | Anal Fissure | Galactosemia | Acute Lung Injury | Greig Cephalopolysyndactyly Syndrome | Hereditary Mixed Polyposis Syndrome | Pantothenate Kinase-associated Neurodegeneration | Osmotic Demyelination Syndrome | Hemosiderosis | Glaucomatocyclitic Crisis | Cryopyrin-associated Periodic Syndromes | Pulmonary Stenosis | Fragile X Syndrome | Lymphangiomatosis | Primary Torsion Dystonia | Mucolipidosis Type II | Hypervalinemia | Pouchitis | Holoprosencephaly | Allan-Herndon-Dudley Syndrome | Corneal Dystrophy | Fibrodysplasia Ossificans Progressiva | Malignant Peripheral Nerve Sheath Tumor | Charcot-Marie-Tooth Disease Axonal Type 2N | Large Granular Lymphocytic Leukemia | Leukoplakia, Oral | Carbohydrate Metabolism Disorders | Craniofacial Dysostosis | Brachial Plexus Neuropathy | Pseudohypoaldosteronism | Knobloch Syndrome | GNE Myopathy | Fibrosarcoma | Dowling-Degos Disease | Bernard-Soulier Syndrome | Charcot-Marie-Tooth Disease Type 4B1 | Nicotine Dependence | Netherton Syndrome | Ichthyosis, X-linked | Neuromyelitis Optica | Multiple Sclerosis, Chronic Progressive | Donnai-Barrow Syndrome | Pseudoexfoliation Syndrome | Epidermolytic Ichthyosis, Annular | Hypocalcemia | Congenital Hereditary Endothelial Dystrophy Type I | Hoyeraal-Hreidarsson Syndrome | Proteasome-associated Autoinflammatory Syndrome 2