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Test your basic knowledge |
Biochemistry Diseases
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Subject
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health-sciences
Instructions:
Answer 50 questions in 15 minutes.
If you are not ready to take this test, you can
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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. Converts adenosine to inosine in purine salvage pathway. Excess ATP - dATP in nucleotide pool = imbalance (feedback inhibition of ribonucleotide reductase - preventing DNA synthesis) - so decreased lymphocyte count (major cause of SCID - severe combi
Adenosine deaminase deficiency
Arsenic toxicity
Hereditary spherocytosis
Fructosouria
2. Aut - dom; mutation on long arm of chr 17 (17 letters in 'von recklinghausen') cafe - au - lait spots - neuronal tumors - Lisch nodules (pigmented iris hamartomas). Skeletal disorders (eg scoliosis) - optic pathway gliomas - pheochromocytoma - increa
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3. Lysosomal storage disease (a sphingolipidosis). Aut - rec deficiency of arylsulfatase A - cerbroside sulfate accumulates. Central / peripheral demyelination - ataxia - dementia.
Hypervitaminosis A
Hereditary spherocytosis
Metachromic leukodystrophy
Patau's syndrome
4. Acquired (e.g. liver dz) or hereditary (urea cycle enzyme deficiencies). inc. NH4 depletes alpha - keotglutarate - inhibiting TCA cycle. Ammonia intox - tremor - slurred speech - somnolence - vomiting - cerebral edema - blurred vision. Tx with benzoa
Cri - du - chat syndrome
Hyperammonemia
Huntington's disease
Down syndrome
5. Type IV familial dyslipidemia. Hepatic overproduction of VLDL; VLDL increased; blood TGs elevated
Biotin deficiency
Neurofibromatosis type I (von Recklinghausen's disease)
Von Gierke's disease
Hypertriglyceridemia
6. 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
Cri - du - chat syndrome
Lesch - Nyhan Syndrome
Cori's disease
Hypophosphatemic rickets
7. Lysosomal alpha -1 -4- glucosidase (acid maltase) deficiency (type II glycogen storage disease. Lysosomal degradation of glycogen impaired (1 -4 = linkages - not branches). Cardiomegaly - systemic findings (liver - muscle) leading to early death. Pom
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8. Brittle bone disease; genetic bone disorder. Variety of gene defects. Type II fatal in utero - neonatal. Most common form: AD with abnormal type I collagen (bone): multiple fractures w/ minimal trauma (e.g. during birth) - blue sclerae (translucent c
Vitamin B5 deficiency
Vitamin D excess
Osteogenesis imperfecta
Familial adenomatous polyposis coli
9. 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
Hypertriglyceridemia
Multiple endocrine neoplasias
Pellagra
Vitamin E deficiency
10. 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
Velocardiofacial syndrome
Prader - Willi syndrome
Abetalipoproteinemia
Cri - du - chat syndrome
11. X- linked trinucleotide repeat disorder (CGG); affects methylation / expression of FMR1 gene - a/w chromosomal breakage. #2 cause of genetic MR (Down's is #1). Macro - orchidism - long face + large jaw - large everted ears - autism.
McArdle's disease
Fragile X
Cystic fibrosis
Duchenne's muscular dystrophy
12. Mitochondrial inheritance (only transmitted via mom); degeneration of retinal ganglion cells / axons; acute loss of central vision
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13. 'Bear liver'guy - arthralgias - fatigue - headaches - skin changes - sore throat - alopecia. Also teratogenic (cleft palate - cardiac abnormalities)
Hypervitaminosis A
Adenosine deaminase deficiency
Leber's hereditary optic neuropathy
Achondroplasia
14. 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
Hypertriglyceridemia
APKD
Duchenne's muscular dystrophy
Velocardiofacial syndrome
15. Facial flushing (niacin 'flushing' in pharm doses for hyperlipidemia treatment).
Angelman's syndrome
Hereditary hemorrhagic telangiectasia (Osler - Weber - Rendu syndrome)
Vitamin B3 excess
Cystinuria
16. Age dep or hereditary lactose intolerance (blacks / Asians); lose brush - border enzyme. Bloating - cramps - osmotic diarrhea. Avoid dairy / use lactase pills.
Lactase deficiency
Ehlers - Danlos syndrome
Hypervitaminosis A
Tay- Sachs disease
17. Microtubule polymerization defect (no lysosomal tracking). Decreased phagocytosis. Recurrent pyogenic infections - partial albinism - peripheral neuropathy.
Hereditary hemorrhagic telangiectasia (Osler - Weber - Rendu syndrome)
Chediak - Higashi syndrome
Albinism
Huntington's disease
18. Protein malnutrition. Skin lesions - edema - liver malfunction (fatty change). Small child - swollen belly.
22q11 deletion syndromes
Albinism
Vitamin D deficiency
Kwashiorkor
19. 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
Vitamin B5 deficiency
Cystic fibrosis
Vitamin E deficiency
Von Gierke's disease
20. 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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21. Cobalamin = B12. macrocytic - megaloblastic anemia. Neuro sx: paresthesias - subacute combined degeneration from abnormal myelin. Prolonged deficiency = irreversible nervous system damage. Found in animal products; synthesized only by microorganisms.
Ornithine transcarbamoylase (OTC) deficiency
Vitamin B12 deficiency
Alkaptonuria (ochronosis)
Phenylketonuria
22. 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
G6PD deficiency
Homocystinuria
Hypophosphatemic rickets
Hyperchylomicronemia
23. Aut - dom cell - signaling defect in fibroblast growth factor receptor 3 (FGFr3). Dwarfism: short limbs but head/trunk normal. A/W advanced paternal age
Cori's disease
Gaucher's disease
Pellagra
Achondroplasia
24. 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
Hypertriglyceridemia
Albinism
Prader - Willi syndrome
Fructose intolerance
25. Aut - rec deficiency of aldolase B; fructose -1- P accumulates; dec. in phosphate = inhibition of glycogenolysis / gluconeogenesis. Hypoglycemia - jaundice - cirrhosis - vomiting. Tx: limit fructose - sucrose (gluc + fruc) intake.
Vitamin E deficiency
Duchenne's muscular dystrophy
Fructose intolerance
Fabry's disease
26. A 22q11 deletion syndrome. Thymic - parathyroid - cardiac defects. Microdeletion at chr 22q11. Due to aberrant development of 3rd / 4th branchial pouches.
DiGeorge syndrome
Vitamin B3 excess
Duchenne's muscular dystrophy
Familial hypercholesterolemia
27. Defects in type IV collagen synthesis; most common form X- linked recessive. Hereditary nephritis - deafness +/- ocular disturbances (type IV collagen important in BM of kidney - ears - eyes)
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28. (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!
Marasmus
Pyruvate dehydrogenase deficiency
Familial adenomatous polyposis coli
Familial hypercholesterolemia
29. Skeletal muscle glycogen phosphorylase deficiency (normally breaks down glycogen to 4- glucose residue branched 'limit dextrans'). Type V glycogen storage disease. Incr. glycogen in mm - but can't break down (mm cramps - myoglobinuria with strenuous
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30. Rickets in children (bending bones); osteomalacia in adults (soft bones). Also hypocalcemic tetany.
Kwashiorkor
von Hippel - Lindau disease
Vitamin D deficiency
Vitamin C (ascorbic acid) deficiency
31. Peroxisomal disease; no alpha - oxidation of branched - chain FA like phytanic acid. Neuro sx; Tx = avoid chlorophyll
Neurofibromatosis type I (von Recklinghausen's disease)
Familial hypercholesterolemia
Refsum disease
22q11 deletion syndromes
32. Hypercalcemia - hypercalciuria - loss of appetite - stupor. Seen in sarcoidosis (inc. activation of vitamin D by epithelioid macrophages).
Fructosouria
Achondroplasia
Cystic fibrosis
Vitamin D excess
33. Trisomy 18 (E=election age) - 1:8000. Severe MR - rockerbottom feet - congenital heart disease. Vs Patau's: micrognathia - low - set ears - clenched hands - prominent occiput. Death w/in 1 yr
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34. Aut - dom; disorder of blood vessels. Telangiectasia - recurrent epistaxis - skin discolorations - AVMs. Incr. in Utah Mormons
Hereditary hemorrhagic telangiectasia (Osler - Weber - Rendu syndrome)
Ehlers - Danlos syndrome
Down syndrome
Prader - Willi syndrome
35. 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
Albinism
Orotic aciduria
Cori's disease
Angelman's syndrome
36. Inclusion cell disease. Can't add mannose -6- P to lysosome proteins. Enzymes secreted outside of cell instead of targeted to lysosome. Lysosomal storage disorder. Coarse facial features - clouded corneas - restricted joint movement - high plasma lev
McArdle's disease
DiGeorge syndrome
Cell disease
Vitamin K deficiency
37. Scurvy - swollen gums - bruising - anemia - poor wound healing. In fruits / vegetables. Facilitates Fe absorption: keeps Fe2+ reduced state - more absorbable. Also involved in hydroxylation of proline - lysine in collagen synthesis; antioxident; need
Vitamin C (ascorbic acid) deficiency
Cri - du - chat syndrome
Von Gierke's disease
Arsenic toxicity
38. 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
Familial adenomatous polyposis coli
Vitamin K deficiency
Hypophosphatemic rickets
Pellagra
39. Cheilosis - corneal vascularization (the 2 C's)
Maple syrup urine disease
Vitamin E deficiency
Vitamin B2 (riboflavin) deficiency
Vitamin K deficiency
40. Aut - rec deficiency of homogentisic acid oxidase (degradative pathway of tyrosine) - benign disease. Dark connective tissue - pigmented sclera - urine turnes black on standing - may have debilitating arthralgias.
22q11 deletion syndromes
Fragile X
Zellweger syndrome
Alkaptonuria (ochronosis)
41. 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
Chediak - Higashi syndrome
Multiple endocrine neoplasias
McArdle's disease
22q11 deletion syndromes
42. Aut - dom - defect in spectrin or ankyrin. Hemolytic anemia - increased MCHC (mean corpuscular hemoglobin concentration). Splenectomy is curative
Arsenic toxicity
Hereditary spherocytosis
Gaucher's disease
Adenosine deaminase deficiency
43. Lysosomal storage disease (a sphingolipidosis). Aut - rec deficiency of sphingomyelinase; sphingomyelin accumulates. Progressive neurodegeneration - hepatosplenomegaly - cherry- red spot on macula - foam cells.
Multiple endocrine neoplasias
Vitamin D excess
Down syndrome
Niemann - Pick disease
44. Pantothenate; in CoA. Dermatitis - enteritis - alopecia - adrenal insufficiency
Vitamin E deficiency
Vitamin D excess
Hereditary spherocytosis
Vitamin B5 deficiency
45. B1 (thiamine) deficiency. Polyneuritis - symmetrical muscle wasting
Vitamin B5 deficiency
Fructose intolerance
Hyperammonemia
Dry beriberi
46. X- linked frame - shift mutation: deletion of dystrophin (DMD anchors mm fibers - esp. skeletal / cardiac. Longest gene: lots of spontaneous mutations). Accel. mm breakdown (weakness in pelvic girdle -> superiorly). Pseudohypertrophy of calf mm (fibr
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47. Aut - rec fructokinase deficiency. Can't P- late fructose - so can't enter cells. Benign - asymptomatic - just fructose in blood - urine.
Edwards' syndrome
Ehlers - Danlos syndrome
Fructosouria
Hereditary hemorrhagic telangiectasia (Osler - Weber - Rendu syndrome)
48. 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).
Fragile X
Pellagra
Classic galactosemia
Marasmus
49. Macrocytic - megaloblastic anemia w/o neuro sx. #1 vitamin deficiency in USA; see in alcoholism / pregnancy (neural tube defects; supplement in early pregnancy) - also drugs (phenytoin - sulfas - MTX). From FOLiage (leafy greens) with small reserve p
Alport's syndrome
Vitamin A deficiency
Folic acid deficiency
Hurler's syndrome
50. X- linked mutated dystrophin. Less severe than Duchenne's. Onset: adolescence - early adulthood.
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