Question

QUESTION 43

1. Answer the following question in no more than 2 well-written sentences.

   Explain ONE reason why Duchenne muscular dystrophy is a complicated genetic disease to treat with gene therapy.

Answer 1

Duchenne muscular dystrophy (DMD) is a relatively common inherited disorder caused by defective expression of the protein dystrophin. The most direct approach to treating this disease would be to restore dystrophin production in muscle.

Of the many approaches being pursued to treat DMD and BMD, gene therapy based on AAV-mediated delivery of microdystrophin is the most direct and promising method to treat the cause of the disorder. The major challenges to this approach are ensuring that microdystrophin can be delivered safely and efficiently without eliciting an immune response.