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Test your basic knowledge |
Biochemistry Diseases
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Study First
Subject
:
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. Aut - dom - defect in spectrin or ankyrin. Hemolytic anemia - increased MCHC (mean corpuscular hemoglobin concentration). Splenectomy is curative
Neurofibromatosis type II
Zellweger syndrome
Marasmus
Hereditary spherocytosis
2. 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.
Vitamin B12 deficiency
Biotin deficiency
Familial adenomatous polyposis coli
McArdle's disease
3. 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
Achondroplasia
Familial adenomatous polyposis coli
Dry beriberi
4. Aut - dom; defective / absent LDL receptor. Elevated LDL. Heterozygotes (1:500) have cholesterol = 300 mg/dL. Homozygotes (rare) chol > 700 mg /dL - severe atherosclerotic dz early in life - tendon xanthomas (classically Achilles); may have MI < 20 y
Familial hypercholesterolemia
Vitamin B6 deficiency
Vitamin C (ascorbic acid) deficiency
Hereditary hemorrhagic telangiectasia (Osler - Weber - Rendu syndrome)
5. Hemolytic anemia (inc fragility of erythrocytes - vit E is antioxidant - protects erythrocytes / membranes from free - radical damage). Muscle weakness - neurodysfunction tooo.
Vitamin E deficiency
Vitamin B3 excess
Zinc deficiency
DiGeorge syndrome
6. 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.
Fructose intolerance
Wet beriberi
Hereditary nonpolyposis colorectal cancer
Prader - Willi syndrome
7. Mutation in nucleotide excision repair proteins (endonucleases that release oligonucleotides containing damaged bases; DNApol + ligase fill in). Can't repair thymidine dimers (UV light). Dry skin with melanoma + other cancers ('children of the night'
Xeroderma pigmentosum
Albinism
Hunter's syndrome
Gaucher's disease
8. Microtubule polymerization defect (no lysosomal tracking). Decreased phagocytosis. Recurrent pyogenic infections - partial albinism - peripheral neuropathy.
Hurler's syndrome
Vitamin E deficiency
Osteogenesis imperfecta
Chediak - Higashi syndrome
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
Hereditary hemorrhagic telangiectasia (Osler - Weber - Rendu syndrome)
Vitamin B6 deficiency
Osteogenesis imperfecta
Homocystinuria
10. 'Bear liver'guy - arthralgias - fatigue - headaches - skin changes - sore throat - alopecia. Also teratogenic (cleft palate - cardiac abnormalities)
Vitamin B5 deficiency
Hypervitaminosis A
Ornithine transcarbamoylase (OTC) deficiency
Fragile X
11. 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
Neurofibromatosis type I (von Recklinghausen's disease)
Adenosine deaminase deficiency
Kartagener's syndrome
Pellagra
12. 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.
Zellweger syndrome
von Hippel - Lindau disease
Fragile X
Albinism
13. Lysosomal storage disease (a sphingolipidosis). Aut - rec deficiency of sphingomyelinase; sphingomyelin accumulates. Progressive neurodegeneration - hepatosplenomegaly - cherry- red spot on macula - foam cells.
Niemann - Pick disease
Hereditary hemorrhagic telangiectasia (Osler - Weber - Rendu syndrome)
Vitamin B6 deficiency
Hypervitaminosis A
14. Delayed wound healing - hypogonadism - dec. adult hair (axillary / facial / pubic); ?predispose to alcoholic cirrhosis?
Osteogenesis imperfecta
Zinc deficiency
Vitamin D deficiency
DiGeorge syndrome
15. (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
Velocardiofacial syndrome
Hyperchylomicronemia
Leber's hereditary optic neuropathy
16. Type IV familial dyslipidemia. Hepatic overproduction of VLDL; VLDL increased; blood TGs elevated
APKD
Galactokinase deficiency
Prader - Willi syndrome
Hypertriglyceridemia
17. Night blindness - dry skin
Vitamin D deficiency
Pyruvate dehydrogenase deficiency
Hyperammonemia
Vitamin A deficiency
18. 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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19. Energy malnutrition. Tissue / muscle wasting - loss of subQ fat - variable edema.
Hurler's syndrome
Marasmus
Vitamin E deficiency
Ornithine transcarbamoylase (OTC) deficiency
20. 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
Niemann - Pick disease
Metachromic leukodystrophy
Familial adenomatous polyposis coli
Fragile X
21. 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
Ehlers - Danlos syndrome
Angelman's syndrome
Vitamin K deficiency
Cell disease
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
Abetalipoproteinemia
G6PD deficiency
Kartagener's syndrome
von Hippel - Lindau disease
23. X- linked mutated dystrophin. Less severe than Duchenne's. Onset: adolescence - early adulthood.
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24. 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
Hyperchylomicronemia
Pyruvate dehydrogenase deficiency
Becker's muscular dystrophy
Ehlers - Danlos syndrome
25. 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 - '
Abetalipoproteinemia
22q11 deletion syndromes
Velocardiofacial syndrome
Neurofibromatosis type II
26. Facial flushing (niacin 'flushing' in pharm doses for hyperlipidemia treatment).
Vitamin B3 excess
Wet beriberi
Adenosine deaminase deficiency
Familial adenomatous polyposis coli
27. X- linked dominant disorder (M/F offspring of affected mom can be affected; all female offspring of affected father diseased). Inc. phosphate wasting at proximal tubule; rickets - like presentation
Cri - du - chat syndrome
Hereditary hemorrhagic telangiectasia (Osler - Weber - Rendu syndrome)
Hypophosphatemic rickets
Tuberous sclerosis
28. Pantothenate; in CoA. Dermatitis - enteritis - alopecia - adrenal insufficiency
Vitamin B5 deficiency
Hereditary hemorrhagic telangiectasia (Osler - Weber - Rendu syndrome)
Hereditary spherocytosis
Prader - Willi syndrome
29. 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
Leber's hereditary optic neuropathy
Hypertriglyceridemia
Albinism
Vitamin D deficiency
30. Mutation in mismatch repair genes (mismatched nucleotides in unmethylated / newly synthesized string recognized + removed)
Achondroplasia
Duchenne's muscular dystrophy
Hereditary nonpolyposis colorectal cancer
Fructose intolerance
31. 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
Achondroplasia
Tuberous sclerosis
Cori's disease
22q11 deletion syndromes
32. Lysosomal storage disease (a sphingolipidosis). X- linked recessive (all other sphingolipidoses AR); deficiency of alpha - galactosidase A ceramide trihexoside accumulates. Peripheral neuropathy (hands / feet) - angiokeratomas - CV / renal disease
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33. 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'
Marasmus
Hyperammonemia
Down syndrome
Tay- Sachs disease
34. Age dep or hereditary lactose intolerance (blacks / Asians); lose brush - border enzyme. Bloating - cramps - osmotic diarrhea. Avoid dairy / use lactase pills.
Multiple endocrine neoplasias
Lactase deficiency
Duchenne's muscular dystrophy
McArdle's disease
35. Aut - rec deficiency of phenylalanine hydroxylase / THB (cofactor) - so can't convert phenylalanine to tyrosine. Results: accumulate phenylalanine (excess phenylketones in urine); tyrosine becomes essential AA. Dx: MR - growth retardation - seizures
Classic galactosemia
Phenylketonuria
Folic acid deficiency
Huntington's disease
36. 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
Tuberous sclerosis
Phenylketonuria
Patau's syndrome
Vitamin C (ascorbic acid) deficiency
37. 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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38. Aut - rec galactokinase deficiency. Galactitol can accumulate if galactose present in diet; relatively mild. Galactose in blood / urine - infantile cataracts (may not track objects - develop social smile.
Galactokinase deficiency
Alkaptonuria (ochronosis)
Vitamin B6 deficiency
Niemann - Pick disease
39. 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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40. 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
Biotin deficiency
Lesch - Nyhan Syndrome
Hereditary nonpolyposis colorectal cancer
Vitamin K deficiency
41. 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
Fructose intolerance
Velocardiofacial syndrome
Cri - du - chat syndrome
Familial adenomatous polyposis coli
42. 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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43. A 22q11 deletion syndrome. Palate - facial - cardiac defects. Microdeletion at chr 22q11. Due to aberrant development of 3rd / 4th branchial pouches.
Fabry's disease
Velocardiofacial syndrome
McArdle's disease
Kwashiorkor
44. 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
Vitamin B5 deficiency
Vitamin D excess
Adenosine deaminase deficiency
Kartagener's syndrome
45. Mitochondrial inheritance (only transmitted via mom); degeneration of retinal ganglion cells / axons; acute loss of central vision
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46. 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
Fabry's disease
Pompe's disease
Folic acid deficiency
Albinism
47. Protein malnutrition. Skin lesions - edema - liver malfunction (fatty change). Small child - swollen belly.
Homocystinuria
Kwashiorkor
G6PD deficiency
Cystinuria
48. 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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49. Lysosomal storage disease (a sphingolipidosis). Aut - rec deficiency of galactocerebosidase; galactocerebroside accumulates. Peripheral neuropathy - developmental delay - optic atrophy - globoid cells (multinucleated).
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50. Aut - dom cell - signaling defect in fibroblast growth factor receptor 3 (FGFr3). Dwarfism: short limbs but head/trunk normal. A/W advanced paternal age
Achondroplasia
Fructose intolerance
Hypertriglyceridemia
Pellagra