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Test your basic knowledge |
Biochemistry Diseases
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Study First
Subject
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health-sciences
Instructions:
Answer 50 questions in 15 minutes.
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Match each statement with the correct term.
Don't refresh. All questions and answers are randomly picked and ordered every time you load a test.
This is a study tool. The 3 wrong answers for each question are randomly chosen from answers to other questions. So, you might find at times the answers obvious, but you will see it re-enforces your understanding as you take the test each time.
1. A 22q11 deletion syndrome. Palate - facial - cardiac defects. Microdeletion at chr 22q11. Due to aberrant development of 3rd / 4th branchial pouches.
Homocystinuria
Velocardiofacial syndrome
Osteogenesis imperfecta
McArdle's disease
2. (Pyruvate + NAD+ + CoA --> acetylCoA + CO2 + NADH; 3 enzymes - requires B1 -2 -3 - CoA - lipoic acid). Pyruvate - alanine accumulate --> lactic acidosis (pyruvate to lactate). Congenital or acquired (e.g. alcoholics - B1 deficiency - lactic acidosis!
Pyruvate dehydrogenase deficiency
Williams syndrome
Down syndrome
Vitamin K deficiency
3. Lysosomal storage disease (a sphingolipidosis). Aut - rec deficiency of arylsulfatase A - cerbroside sulfate accumulates. Central / peripheral demyelination - ataxia - dementia.
Gaucher's disease
Metachromic leukodystrophy
Kwashiorkor
Ehlers - Danlos syndrome
4. A 22q11 deletion syndrome. Thymic - parathyroid - cardiac defects. Microdeletion at chr 22q11. Due to aberrant development of 3rd / 4th branchial pouches.
DiGeorge syndrome
Vitamin B2 (riboflavin) deficiency
Dry beriberi
Fructose intolerance
5. Rickets in children (bending bones); osteomalacia in adults (soft bones). Also hypocalcemic tetany.
Vitamin D deficiency
Tay- Sachs disease
Hurler's syndrome
Ornithine transcarbamoylase (OTC) deficiency
6. Energy malnutrition. Tissue / muscle wasting - loss of subQ fat - variable edema.
Marasmus
Vitamin C (ascorbic acid) deficiency
Lesch - Nyhan Syndrome
Familial hypercholesterolemia
7. Mitochondrial inheritance (only transmitted via mom); degeneration of retinal ganglion cells / axons; acute loss of central vision
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8. Lysosomal storage disease (a sphingolipidosis) - Aut - rec deficiency of beta - glucocerebrosidase; glucocerebroside accumulates. Hepatosplenomegaly - aseptic necrosis of femur - bone crises - Gaucher's cells (macrophages that look like crumpled tiss
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9. Aut - rec - 3 forms. All interfere with conversion of methionine to cysteine - cysteine becomes essential; homocysteine accumulates. Causes: 1) cystathionine synthase deficiency (Tx: dec Met - inc. Cys intake. Inc B12 / folate in diet to drive conver
Familial hypercholesterolemia
Hypervitaminosis A
Homocystinuria
Familial hypercholesterolemia
10. Severe vitamin B3 (niacin) deficiency (less severe = glossitis). Diarrhea - dementia - dermatitis. Can be caused by Hartnup disease (dec. tryptophan absorption) - malignant carcinoid syndrome (inc tryptophan metabolism) (B3 made from tryptophan) and
Pellagra
Hypertriglyceridemia
G6PD deficiency
Gaucher's disease
11. X- linked rec; most common urea cycle disorder (others aut - rec). Can't eliminate ammonia; often dx'd perinatally but can be later. Dx: orotic acid in blood / urine (excess carbamoyl phosphate converted to orotic acid) - dec. BUN - hyperammonemia sx
Lactase deficiency
Vitamin B5 deficiency
Zinc deficiency
Ornithine transcarbamoylase (OTC) deficiency
12. X- linked recessive - absence of HGPRT (hypoxanthine to IMP - guanine to GMP in purine salvage pathway). Can't salvage purines - so increased elimination = incr uric acid production. Retardation - self - mutilation - aggression - hyperuricemia - gout
Lesch - Nyhan Syndrome
Vitamin D excess
Kartagener's syndrome
DiGeorge syndrome
13. Cheilosis - corneal vascularization (the 2 C's)
Alport's syndrome
Vitamin B2 (riboflavin) deficiency
Phenylketonuria
Cystic fibrosis
14. Aut - rec absence of galactose -1- phosphate uridyltransferase; toxic substances accumulate (e.g. galactitol in lens of eye). FTT - jaundice - hepatomegaly - infantile cataracts - MR. Tx: no galactose / lactose (glucose + galactose).
Classic galactosemia
Ehlers - Danlos syndrome
von Hippel - Lindau disease
Pellagra
15. Glucose -6- phosphatase deficiency (type I glycogen storage disease). Can't de - plate G6P (glycogenolysis & gluconeogenesis). Severe fasting hypoglycemia - inc. glycogen in liver - inc. blood lactate (gluconeogenesis impaired) - hepatomegaly.
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16. Aut - dom; disorder of blood vessels. Telangiectasia - recurrent epistaxis - skin discolorations - AVMs. Incr. in Utah Mormons
Familial hypercholesterolemia
von Hippel - Lindau disease
Hereditary hemorrhagic telangiectasia (Osler - Weber - Rendu syndrome)
Ornithine transcarbamoylase (OTC) deficiency
17. Peroxisomal disease; no alpha - oxidation of branched - chain FA like phytanic acid. Neuro sx; Tx = avoid chlorophyll
Ornithine transcarbamoylase (OTC) deficiency
Hyperchylomicronemia
Hereditary hemorrhagic telangiectasia (Osler - Weber - Rendu syndrome)
Refsum disease
18. Type IV familial dyslipidemia. Hepatic overproduction of VLDL; VLDL increased; blood TGs elevated
Hypertriglyceridemia
Kartagener's syndrome
Marasmus
Biotin deficiency
19. Aut - dom. Facial lesions (adenoma sebaceum) - hypopigmented 'ash - leaf spots' on skin - cortical / retinal hamartomas - seizures - MR - renal cysts - renal angiomyolipomas - cardiac rhabdomyomas. Inc. incidence astrocytomas. Incomplete penetrance w
Vitamin D excess
Tuberous sclerosis
Cell disease
Wet beriberi
20. Trisomy 13 (P = puberty age) - 1:15 -000. Severe MR - rockerbottom feet - congenital heart disease. Vs. Edwards': cleft lip / Palate - holoProsencephaly - Polydactyly (P's) - micropthalmia - microcephaly. Death w/in 1 yr
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21. X- linked recessive. G6PD: G6P + NADP+ -> 6PG + NADPH; NADPH needed to reduce GSSG to 2GSH (glutathione reductase) for H2O2 to 2H2O conversion. Only pathway for making reduced GSH in RBC - so can't detoxify free radicals / peroxides (fava beans - sul
Xeroderma pigmentosum
Hypertriglyceridemia
Huntington's disease
G6PD deficiency
22. Type I familial dyslipidemia. Lipoprotein lipase deficiency or altered apolipoprotein C- II (LPL cofactor) = can't degrade TG circulating in chylomicrons - VLDLs. Increased chylomicrons - elevated cholesterol / TGs in blood
Alkaptonuria (ochronosis)
Vitamin B3 excess
Duchenne's muscular dystrophy
Hyperchylomicronemia
23. Facial flushing (niacin 'flushing' in pharm doses for hyperlipidemia treatment).
Albinism
Vitamin B3 excess
Von Gierke's disease
Velocardiofacial syndrome
24. Aut - rec - defect in renal tubular AA transporter (cysteine - ornithine - lysine - arginine in renal PCT). inc. cystine (2 cysteines w/ disulfide bond) in urine --> cystine kidney stones (cystine staghorn calculi). Common (1:7000); Tx with acetazola
Cystinuria
Vitamin B12 deficiency
Dry beriberi
McArdle's disease
25. Night blindness - dry skin
DiGeorge syndrome
Vitamin A deficiency
Zellweger syndrome
Zinc deficiency
26. Pyridoxine = B6. Convulsions - hyperirritability - peripheral neuropathy. Deficiency inducible by INH - oral contraceptives. Converted to pyridoxal phosphate: used in transamination (ALT - AST - etc) - decarboxylation - glycogen phosphorylase - heme
Cystinuria
Vitamin B6 deficiency
McArdle's disease
Familial adenomatous polyposis coli
27. X- linked mutated dystrophin. Less severe than Duchenne's. Onset: adolescence - early adulthood.
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28. Aut - rec; can't convert orotic acid to UMP in de novo pyrimidine synthesis pathway (defect in orotic acid PRT or orotidine 5''- P- decarboxylase. Incr. orotic acid in urine - megaloblastic anemia (that doesn't get better with B12 / folate supplement
Orotic aciduria
Wet beriberi
Pellagra
Chediak - Higashi syndrome
29. Lysosomal storage disease (a mucopolysaccharidosis). Aut - rec deficiency of alpha - L- iduronidase; heparan sulfate - dermatan sulfate accumulate. Developmental delay - gargoylism (thickened gingiva + upturned nose) - airway obstruction - corneal cl
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30. Adult polycystic kidney disease. Aut - dom - 90% from mutation in APKD1 - chr 16 (16 letters in 'polycystic kidney'). Always bilateral - big kidneys (lots of big cysts). Flank pain - hematuria - HTN - progressive renal failure. a/w polycystic liver d
Cori's disease
Pyruvate dehydrogenase deficiency
APKD
Adenosine deaminase deficiency
31. Faulty collagen synthesis: hyperextensible skin - tendency to bleed (easy bruising) - hypermobile joints. Mostly type III collagen. 6 types - inheritance / severity vary (AD/AR). A/w joint dislocation - berry aneurysms - organ rupture
Ehlers - Danlos syndrome
Lesch - Nyhan Syndrome
22q11 deletion syndromes
Kartagener's syndrome
32. Lysosomal storage disease (a sphingolipidosis). Aut - rec deficiency of hexosaminidase A; GM2 ganglioside accumulates. Progressive neurodegeneration - developmental delay - cherry- red spot on macula - lysosomes with 'onion skin'
Hereditary spherocytosis
Osteogenesis imperfecta
Marasmus
Tay- Sachs disease
33. Peroxisomal disease; can't metabolize very long chain FA (VLCFAs) or branched - chain FAs (e.g. phytanic acid - via alpha - oxidation). Can't form myelin in CNS. Hypotonia - seizures - hepatomegaly - MR - early death.
McArdle's disease
Vitamin D excess
Zellweger syndrome
Vitamin E deficiency
34. Aut - dom; deletion of VHL gene (tumor suppressor) on chr 3 (3 words for chr 3). Results in constitutive expression of HIF (transcription factor) - activation of angiogenic growth factors. Hemangioblastomas of retina / cerebellum / medulla - 50% get
von Hippel - Lindau disease
Velocardiofacial syndrome
Hereditary hemorrhagic telangiectasia (Osler - Weber - Rendu syndrome)
Niemann - Pick disease
35. Age dep or hereditary lactose intolerance (blacks / Asians); lose brush - border enzyme. Bloating - cramps - osmotic diarrhea. Avoid dairy / use lactase pills.
Neurofibromatosis type II
Lactase deficiency
Fructose intolerance
Zellweger syndrome
36. Variable presentation - incl CATCH-22: Cleft palate - Abnormal facies - Thymic aplasia / T- cell deficiency - Cardiac defects - Hypocalcemia (2ary to parathyroid aplasia) - from microdeletion at chr 22q11. Due to aberrant development of 3rd / 4th bra
Neurofibromatosis type I (von Recklinghausen's disease)
22q11 deletion syndromes
Xeroderma pigmentosum
Familial hypercholesterolemia
37. Variable inheritance (locus heterogeneity); vs ocular albinism (x- linked recessive). Congenital deficiency of either 1) tyrosinase (can't make melanin from tyrosine; aut - rec); 2) defective tyrosine transporters (dec. tyrosine = dec. melanin); can
Ehlers - Danlos syndrome
Adenosine deaminase deficiency
Albinism
APKD
38. Aut - rec (CFTR gene - chr 7; CFTR channel secretes Cl - in lungs / GI - resorbes Cl - from sweat). Thick mucus - plugs lungs - pancreas - liver. Recurrent pulm infections (Pseudomonas - S aureus) - chronic bronchitis - bronchiectasis - pancreatic in
Cystic fibrosis
Duchenne's muscular dystrophy
Kartagener's syndrome
Phenylketonuria
39. Bleeds (gamma - carboxylation of glutamic acid residues on factors II - VII - IX - X - protein C - S). Neonatal hemorrhage (inc PT - aPTT - normal bleeding time - sterile intestine in newborn - can't synthesize vitK - synthesized by intestinal flora
Multiple endocrine neoplasias
Vitamin K deficiency
Familial adenomatous polyposis coli
McArdle's disease
40. Lysosomal storage disease (a mucopolysaccharidosis). X- linked rec deficiency of iduronate sulfatase; heparan sulfate - dermatan sulfate accumulate. Mild Hurler's phenotype (developmental delay - gargoylism (thickened gingiva + upturned nose) - airwa
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41. Chr 15 - normally active maternal (AngelMan = maternal) allele deleted. MR - seizures ataxia - inappropriate laughter ('happy puppet'). Other parent's allele inactivated / imprinted by methylation; can also be from uniparental disomy (2 copies of chr
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42. Aut - dom; I/II/III - familial tumors of endocrine glands (pancreas - parathyroid - pituitary - thyroid - adrenal medulla). II / III a/w ret gene
Multiple endocrine neoplasias
Neurofibromatosis type I (von Recklinghausen's disease)
Familial hypercholesterolemia
Pyruvate dehydrogenase deficiency
43. Chr 15 - normally active parental (Prader = parental) allele deleted. MR - hyperphagia - obesity - hypogonadism - hypotonia. Other parent's allele inactivated / imprinted by methylation; can also be from uniparental disomy (2 copies of chr from 1 par
Tuberous sclerosis
Prader - Willi syndrome
Hereditary spherocytosis
Vitamin B12 deficiency
44. Microdeletion of short arm of chr 5: 46 -XX or XY - 5p -. Microcephaly - moderate/severe MR - high pitched crying/mewing (cry of the cat) - epicanthal folds - cardiac abnormalities
Krabbe's disease
Cri - du - chat syndrome
Tuberous sclerosis
Albinism
45. Protein malnutrition. Skin lesions - edema - liver malfunction (fatty change). Small child - swollen belly.
Vitamin C (ascorbic acid) deficiency
Classic galactosemia
Hurler's syndrome
Kwashiorkor
46. Delayed wound healing - hypogonadism - dec. adult hair (axillary / facial / pubic); ?predispose to alcoholic cirrhosis?
Angelman's syndrome
DiGeorge syndrome
Zinc deficiency
Gaucher's disease
47. Aut - dom - Fibrillin defect (scaffolding for elastin formation - stretchy protein for lungs - large arteries - elastic ligaments - vocal cords - ligamenta flava). Connective tissue disorder: skeleton - heart - eyes affected. Tall - long extremities
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48. Aut - rec deficiency in apoB-100 (binds LDL receptor; mediates VLDL secretion) and apoB-48 (mediates chylomicron secretion) - so can't synthesize lipoproteins. Sx appear in 1st months of life: FTT - steatorrhea - acanthocytosis (= RBC with spikes - '
Vitamin B2 (riboflavin) deficiency
Abetalipoproteinemia
Angelman's syndrome
Pellagra
49. Aut - dom; mutation of huntingtin (chr 4 - 'hunting 4 food') - CAG repeats. Depression - progressive dementia - choreiform movements - caudate atrophy; manifests 20-50 y/o. Dec GABA - ACh in brain.
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50. Aut - dom; mutation in APC gene (chr 5 - 'polyp' has 5 letters). Colon covered with adenomatous polyps after puberty. Progresses to colon cancer unless resected
Kartagener's syndrome
Angelman's syndrome
Familial adenomatous polyposis coli
Pyruvate dehydrogenase deficiency