| Gaucher's Disease | |||||||
| Cluster of autosomal recessive disorders Mutations in gene encoding protein glucocerebrosidase Most common Lysosomal storage disorder Glucocerebrosidase - enzyme that cleaves glucose residue from ceramide. As a result it accumulates in phagocytes and sometimes in CNS Glucocrebrosidase continuously formed from catabolism of glycoproteins from mature cells of senescent leukocytes and erythrocytes 3 types of Gaucher Disease Type 1: storage of glucocerebrosidase linked to mononuclear phagocytes, Brain not involved, splenic and skeletal involvement, reduced glucocerebrosidase activity Type 2: no activity of glucocerebrosidase, hepatomegaly, CNS involved Type 3: Combination of Type 1 and Type 2, in infants |
|||||||
| Gaucher cells: phagocyte filled with glucocerebrosidase found in liver, spleen, bone marrow, lymph nodes, tonsillar thymus, peyes patches. | |||||||
| Cells are PAS positive | |||||||
| Symptoms: | |||||||
| Enlarged Spleen Lymphadenopathy Accumulation of Gaucher Cells in bone marrow Gaucher cells in Virchow-Robbins spaces Arterioles surounded by swollen adventitial cells Pancytopenia and thrombus |
|||||||
| Index Personal MedBox | |||||||