500
Name the diseases associated with the following:
1) Inherited deficiency of the lysosomal enzyme galactocerebroside B-galactosidase, characterized by extensive CNS/PNS demyelination and the presence of multi-nucleated macrophages in the cerebral white patter? 2) The gene defect chromosome 10 encoding the peroxisomal enzyme phytanoyl-CoA-hydroxylase, cardinal manifestations include pigmentary retinal degeneration, chronic hypertrophic neuropathy, ataxia, and other cerebellar signs (nystagmus and intention tremor).
3) AR disorder due to mutations in the gene that encodes MTP (microsomal triglyceride transfer protein) on chromosome 4, clinical present with ataxia, neuropathy, retinitis pigmentosa, acanthocytosis.
4) AR disorder characterized by severe deficiency of plasma alpha or high-density lipoproteins resulting in deposition of cholesterol esters in many tissues including the reticuloendothelial system and peripheral nerves.
5) AR disorder of sulfatide metabolism caused by deficiency of the lysosomal enzyme, arylsulfatase A and subsequent accumulation of sulfatides in brain, peripheral nerves and other tissues.
1. Globoid cell leukodystrophy (Krabbe disease).
2. Refsum’s disease.
3. Abetalipoproteinemia/ Bassen-Kornweig syndrome.
4. Tangier disease.
5. Metachromatic Leukodystrophy.