Hurler syndrome is a rare recessive gene6cdisorder due to a deficiency of laroni

Question

Hurler syndrome is a rare recessive gene6cdisorder due to a

deficiency of laronidase enzyme. The syndromehas many

symptoms, and organ failure may lead to death.AldurazymeR

(laronidase) enzyme replacement therapy canreduce some of the

symptoms. Which of the following best describesthe basis of this

treatment?

A. It changes thepa6ent’s genotype from homozygous to

heterozygous.

B. It providesfunc6onal laronidase protein which the pa6entis

unable to express as agene product.

C. It allows the pa6entto produce func6onal laronidase protein.

D. It changes thedominance of the pa6ent’s allelesfrom

complete recessive to acodominant rela6onship.

E. More than one of theabove statements in A-D are correct.

Explanation / Answer

the answer is B. the enzyme replacement therapy simply gives the patient whathe is not able to produce. it is not a gene therapy but only a replacement therapy. so the patient is given the treatment every week for the restof his life.the patient cannot produce this enzyme even aftertreatment. so the answer is that the treatmentprovides functional laronidase protein which the patient is unableto express as a gene product.

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