Leukodystrophies and Gene Therapy Quiz

What is the primary function of the myelin sheath in the nervous system?

• To insulate and protect nerve cells

• To store genetic information

• To transmit electrical signals

• To produce neurotransmitters

Which faulty gene is associated with Adrenoleukodystrophy (ALD)?

• ABCD1

• Beryl sulphate ace a

• CFTR

• BRCA1

What is the role of the Beryl sulphate ace a gene in Metachromatic Leukodystrophy (MLD)?

• To produce a protein that breaks down fatty acids

• To produce a protein that helps clean up the cell

• To produce a protein that transmits signals

• To produce a protein that insulates nerve cells

In the ex vivo gene therapy approach, what is the purpose of using a vector?

• To destroy accumulated fatty acids

• To stimulate the production of new stem cells

• To deliver a working copy of the disease-causing gene

• To remove faulty genes from the patient's cells

What is the ultimate goal of the gene therapy being developed for leukodystrophies?

• To eliminate all symptoms immediately

• To completely cure the disease

• To halt disease progression and restore protein function

• To replace all damaged nerve cells