A 3-year-old boy presents with failure to thrive, fatty stools, and cholestasis. Laboratory testing reveals severely elevated triglycerides (1200 mg/dL), very low cholesterol (80 mg/dL), and fat-soluble vitamin deficiencies. Genetic testing reveals mutations in the microsomal triglyceride transfer protein (MTP) gene. Which of the following pathophysiologic processes is most directly impaired?

  1. A)Conversion of cholesterol to pregnenolone
  2. B)Hepatic cholesterol uptake via LDL receptor
  3. C)Lipoprotein lipase-mediated hydrolysis of triglycerides
  4. D)Intestinal cholesterol absorption in the terminal ileum
  5. E)Apolipoprotein B-100 lipidation and VLDL assemblyGABARITO

Explicação

This patient has abetalipoproteinemia, caused by MTP gene mutations. MTP (microsomal triglyceride transfer protein) is essential for lipidating apoB-100 in hepatocytes (VLDL assembly) and apoB-48 in enterocytes (chylomicron assembly). Without functional MTP, a... Ver explicação completa e trilha adaptativa →

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