4. Describe the genetics of Duchenne muscular dystrophy. 5. CRISPR is a technology that we can...

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?

Expert Solution

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

Sydney Mullin answered 1 year ago

The nurse is caring for a child that has been diagnosed with Duchenne Muscular Dystrophy (DMD)....

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Fill in the blank. Duchenne muscular dystrophy is caused by an X-linked recessive mutation in the...

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Outline a workflow for the diagnosis of Duchenne Muscular Dystrophy. Include what patient details and material...

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A patient with Duchenne muscular dystrophy presents with fibrosis, where some of the contractile muscle fibers are replaced with scar tissue, although the nervous system and neuromuscular junction are not altered. In this patient the sarcomere length, muscle belly length, and fiber type distribution are equivalent to a control subject. A. What would be the predicted change in maximum isometric force and maximum unloaded shortening velocity compared to a control subject. Describe using terms of muscle architecture (sarcomere arrangement- parallel...

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3. Why is carrier status for Duchenne muscular dystrophy (DMD) usually not transmitted from affected fathers...

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The “reading frame hypothesis” can explain the difference in phenotype between Duchenne and Becker muscular dystrophies...

The “reading frame hypothesis” can explain the difference in phenotype between Duchenne and Becker muscular dystrophies (DMD and BMD respectively), diseases which are both caused by mutations affecting the gene encoding the protein dystrophin. How has our understanding of the molecular bases of these two diseases led to development of molecular therapies for DMD using antisense oligonucleotides to modulate dystrophin RNA splicing, and involving a mechanism of so-called exon skipping? (min 500words)

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