Question

Question 1: Offspring with Pompe are cured for life once they receive a dose of the GAA enzyme medicine.

True or False?

Question 2: What part of the body is affected by Pompe Disease?
Brain and Heart
Heart and muscles
Muscles and stomach
liver and heart


Question 3: What part of the cells are involved with Pompe Disease?
Mitochondria
cytoplasm
ribosome
lysosome


Question 4: A codon is made up of____ letters
2
3
4
as many as needed to code for the correct amino acid

Question 5: Where is lots of glycogen stored in the body?
Heart muscle
skeletal muscles
respiratory muscles
all of the above

Answer 1

1. The given statement is False

The Pompe disease is usually treated with the help of enzyme replacement therapy in which recombinant enzyme is being administered in the patient at regular intervals.

2. The correct answer would be heart and muscles.

Pompe disease is caused by a mutation in a gene responsible for the synthesis of GAA (d acid alpha-glucosidase), an enzyme responsible for the breakdown of the glycogen in the muscles.

Due to this defect, the glycogen gets accumulated in the body, however, muscles and heart are the most affected parts.

3. The correct answer would be lysosomes.

GAA enzyme performs its functions in lysosomes.

4. The correct answer would be 3.

A codon is made up of three nucleotides which code for a specific amino acid.

5. The correct answer would be skeletal muscles.

In the human body, the glycogen is mainly stored in skeletal muscle and liver.