Muscular Dystrophy

Question 1

What is muscular dystrophy

Answer 1

A fatal muscle wasting disease primarily strikes boys

Question 2

What are symptoms of muscular dystrophy

Answer 2

Progressive weakness
At about 2-3 years sees signs

Wheelchair bound by 10-12

Dies by 20 by either respiratory failure or from heart failure

Question 3

What causes muscular dystrophy

Answer 3

A recessive genetic defect on the X sex chromosomes

If male gets an X from mother who has the Carrier the son will get it

Question 4

What is dystrophin

Answer 4

A large protein that provides structural stability to the muscle cells plasma membrane

Part of a complex membrane protein

Dystrophic muscles mean they lack dystrophin

Question 5

What is the gene therapy approach

Answer 5

Healthy genes are delivered to the defected cells by means of viruses

The desired gene is inserted like a virus to change the codes of the affected cells

Question 6

What is the cell transplant approach

Answer 6

Injecting cells that can functionally respects the dystrophic muscle tissue

Myoblasts

Question 7

What is the utrophin approach

Answer 7

The utrophin upregulation compensates for the lack of dystrophin protein

Question 8

What is utrophin

Answer 8

A naturally occurring protein that is nearly identical to dystrophin

Question 9

What is the anti myostatin approach

Answer 9

Myostatin is a protein produced in muscle cells that works to inhibit skeletal muscle growth

Working on how to use this to help the disease