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Lysosomal storage diseases
Sphingolipids
- Derived from sphingosine
- Ceramide
- The parent compound for other sphingolipids.
- Fabry's disease: lack α-galactosidase A, accumulates ceramide trihexoside. Peripheral neuropathy, angiokeratomas, cardiovascular/renal disease.
- Sphingomyelin
- Most common sphingolipid = ceramide + phosphocholine or phosphoethanolamine.
- Niemann-Pick disease: lack sphingomyelinase, accumulates sphingomyelin. A type of leukodystrophy. Progressive neurodegeneration, hepatosplenomegaly, cherry-red spot on macula, foam cells.
- Cerebroside
- Galactocerebrosides are found in brain neurons.
- Krabbe's disease: lack galactocerebrosidase, accumulate galactocerebroside. A type of leukodystrophy, disrupts myelin sheath. Peripheral neuropathy, developmental delay, optic atrophy, globoid cells.
- Sulfate at 3 position of galactose form cerebroside sulfate.
- Metachromatic leukodystrophy: lack arylsulfatase A, accumulate cerebroside sulfate, disrupt myelin sheath. Central and peripheral demyelination with ataxia, muscle wasting, dementia.
- Glucocerebrosides are found elsewhere.
- Gaucher's disease: lack β-glucocerebrosidase, accumulate glucocerebroside. Hepatosplenomegaly, aseptic necrosis of femur, bone crisis, Gaucher's cells. In less severe cases, patients fatigue and bruise easily. In severe cases, there's extensive brain damage.
- Ganglioside
- Sphingoglycolipids with sialic acid (N-acetylneuraminic acid) as one of its sugar residues.
- Most prevalent in brain.
- Tay Sachs: lack hexosaminidase A, accumulate GM2 ganglioside. Progressive neurodegeneration, developmental delay, cherry-red spot on macula, lysosomes with onion skin. Prevalent in Eastern European and Askhenazi Jewish population.
Oligosaccharides and Polysaccharides
- Sugars are joined by the glycoside bond (acetal in organic chemistry) to make oligosaccharides.
- Lactose = galactose + glucose (β 1-4 linkage).
- Lactose synthase: UDP-galactose + glucose → Lactose + UDP
- Secreted in milk.
- Broken down by lactase.
- Sucrose = glucose + fructose (α 1-2)
- Maltose = glucose + glucose (α 1-4). It's basically starch broken down into disaccharide.
- Isomaltose = maltose but with α 1-6 linkage.
- Cellobiose = glucose + glucose (β 1-4). It's basically cellulose broken down into disaccharide. We can't digest cellulose or cellobiose.
- Starch
- Amylose = glucose α 1-4 polymer. Broken down by amylase.
- Amylopectin = amylose with α 1-6 branches. Debranching enzyme needed to break α 1-6 linkage.
- Glycogen = more branched than amylopectin = glucose α 1-4 polymer with α 1-6 branches.
- Broken down by phosphorylase and debranching enzyme.
- Made by synthase and branching enzyme.
- Type II glycogen storage disease, aka Pompe's disease = lack lysosomal α 1-4 glucosidase.
- Plant polymers
- Cellulose = glucose β 1-4 polymer. Found in fiber. Broken down by cellulase, but we don't have this enzyme.
- Pectin = galacturonic acid α 1-4 polymer. Extracted from plants, used in gelling agents, like jelly.
- Chitin = N-acetylglucosamine β 1-4 polymer. Found in mushrooms and the shells of shrimps and lobsters.
- Peptidoglycan = bacteria cell wall
- N-acetylglucosamine + N-acetylmuramic acid (β 1-4).
- Cross-linked by amino acids chains.
- Glycosaminoglycan = mucopolysaccharides = polymer of amino sugar with sulfate/carboxylate.
- Found in ground substance (gel-like matrix) of connective tissue.
- Chondroitin sulfate and keratan sulfate are components of connective tissue. Chondroitin sulfate and glucosamine sulfate are used to treat damaged cartilage.
- Heparin = anticoagulent. Found in mast cells lining arterial walls.
- Hyaluronic acid = vitreous humor and synovial fluid. Degraded by hyaluronidase.
- Dermatan = found in skin.
- Hurler's syndrome: lack α-L-iduronidase, accumulate Heparan sulfate, dermatan sulfate. Developmental delay, gargoylism, airway obstruction, corneal clouding, hepatosplenomegaly.
- Hunter's syndrome: lack iduronate sulfatase, accumulate heparan sulfate, dermatan sulfate. Mild Hurler's syndrome.
Glycoproteins
- Antibodies.
- Antigenic determinants
- ABO blood group is due to oligosaccharide differences on the cell surface glycoproteins.
- N-linked: glycoside (acetal) bond of sugar to asparagine.
- Cotranslational synthesis
- Link oligosaccharide to lipid (dolichol).
- Transfer oligosaccharide to translating peptide by oligosaccharyltransferase.
- Remove some sugar units.
- Add some sugar units.
- O-linked: glycoside (acetal) bond of sugar to serine of threonine.
- Posttranslational synthesis
- Addition of sugars in the Golgi
- Starts off with addition of N-acetylgalactosamine (GalNAc) by GalNAc transferase.
- 5-Hydroxylysine in collagen.
- GPI anchor = C-term of peptide + phosphoethanolamine + core tetrasaccharide + phosphatidylinositol = anchors protein to membrane.
Proteoglycans
- Core protein + glycosaminoglycan chains.
- High carbohydrate content (~90% by weight).
- Sugars added as this glycoprotein travels through the golgi network.
- Deficiency in lysosomal enzymes lead to accumulation of proteoglycans, which lead to Hurler's syndrome.
- Cartilage proteoglycan = core protein + chondroitin = gives resilience.
Eicosanoids
Cholesterol Biosynthesis
Phospholipids
- Aka. glycerophospholipids or phosphoglycerides.
- Found in cell membranes.
- Plasmalogen = phospholipid with unsaturated ether linkage at C1 (opposite to phosphate carbon) instead of an ester.
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