Lesson 11: CRISPR Treats DMD in Mice Article

What is Duchenne muscular dystrophy (DMD)?

• A genetic disease affecting muscle function

• A type of cancer

• A viral infection

• A bacterial infection

Tags

NGSS.HS-LS1-1

NGSS.HS-LS3-1

NGSS.HS-LS3-2

What gene editing technology is mentioned as a potential therapy for DMD?

• CRISPR/Cas9

• RNA interference

• Gene splicing

• Polymerase chain reaction

What is the primary goal of gene editing in the context of DMD?

• To increase muscle mass

• To restore expression of a modified dystrophin gene

• To enhance immune response

• To reduce body fat

Tags

NGSS.HS-LS1-1

What percentage of DMD patients could potentially benefit from exon-skipping therapies?

• 13%

• 50%

• 83%

• 100%

What is the purpose of using CRISPR/Cas9 in the mdx mouse model of DMD?

• To increase muscle mass.

• To excise exon 23 from the dystrophin gene.

• To enhance cardiac function.

• To promote cell division.

Tags

NGSS.HS-LS1-1

What was observed in the Western blot of whole muscle lysates in the study?

• No change in dystrophin levels.

• Increase in dystrophin to ~8% of normal levels.

• Complete restoration of dystrophin.

• Decrease in dystrophin levels.

What is the role of dystrophin in muscle cells?

• To promote cell division.

• To link the cytoskeleton and the extracellular matrix.

• To increase protein synthesis.

• To enhance DNA replication.

What was the observed effect of CRISPR/Cas9 treatment on muscle tissue in the study?

• Increased fibrosis and inflammation

• Decreased macrophages and neutrophils

• No change in muscle morphology

• Increased muscle degeneration

What improvement was noted in Cas9/gRNA-treated muscle regarding contraction injury?

• 20% of initial force recovery

• 50% of initial force recovery

• 70% of initial force recovery

• No recovery observed

What is the potential benefit of using a single gene editing treatment for DMD?

• It can only treat cardiac muscle

• It has no effect on muscle function

• It provides a single, general treatment rather than needing unique treatments for each individual

• It is ineffective in treating genetic diseases