Question

4. Describe the genetics of Duchenne muscular dystrophy.

5. CRISPR is a technology that we can use to target a specific location on the DNA. How can CRISPR be used on a mutant DMD gene?

Answer 1

Answer 4 ) Duchenne muscular dystrophy is a inherited disorder because of the mutated DMD gene which fails to produce dystrophin protein . Dystrophin gene is located in the short arm of X chromosome in Xp21.2 locus . Mutation in dystrophin gene can be deletion of one or more parts of it . the function of dystrophin protein is to transfer the force of muscle contraction from inside of muscle cell to cell membrane. Actions of dystrophin causes formation of fibrous tissue in muscle and there by causes damage and weakness in muscle. Since it a X linked recessive disorder females will be carriers and males would be affected individual .

Answer 5 ) CRISPR technology is a genome editing technology in genetic engineering. Through CRISPR, deletion or excision of intragenic DNA can be done along with correction DMD Gene which helps to restore expression of dystrophin protein . In this technology patients with Duchenne muscular dystrophy treated by taking out duplicated exons ,accurate correction of causative mutation by HDR based pathway and induce the expression of protein like utrophin