Understanding Duchenne Muscular Dystrophy and Gene Therapy

What is the primary reason Duchenne muscular dystrophy affects mostly boys?

• It is linked to the Y chromosome.

• Boys have a stronger immune response.

• It is an autosomal recessive disorder.

• The dystrophin gene is located on the X chromosome.

What is the main goal of gene therapy in treating Duchenne muscular dystrophy?

• To completely cure the disease.

• To introduce functional genes to slow disease progression.

• To enhance the immune system.

• To replace all muscle tissues.

Why is the dystrophin gene challenging to use in gene therapy?

• It mutates too quickly.

• It is the largest known gene and difficult to package.

• It is too small to be effective.

• It is not compatible with human cells.

What is a vector in the context of gene therapy?

• A virus-derived tool to deliver genes.

• A type of muscle protein.

• A symptom of Duchenne muscular dystrophy.

• A genetic disorder.

What is one of the potential benefits of gene editing in treating Duchenne muscular dystrophy?

• It can increase the number of muscle cells.

• It can completely eliminate the disease.

• It can prevent all genetic disorders.

• It can directly modify genes within muscle cells.