Is 2'-O-methyl PS still used in exon skipping for Duchenne muscular dystrophy?

Expert Solution

Ans- Duchenne muscular dystrophy (DMD) is the most common inherited muscular disease in childhood mostly affected boys. DMD disease is caused by mutations in the dystrophin gene. dystrophin gene encodes a dystrophin protein of muscle cells, dystrophin proteins helps in strengthen muscle fibers and protect them from injury. Exon skipping therapy is useful to recover dystrophin expression in DMD. Antisense oligonucleotides (AOs) are used in exon skipping therapy. Initiation of exon skipping with antisense oligonucleotides (AOs) that convert out -of- frame dystrophin mRNA into in-frame sequences. Exon deletion mutations maintain the original reading frame in the mRNA. 2'-O-methyl Phosphorothioate is an example of antisense oligonucleotide (AOs) that are used in exon skipping for Duchenne muscular dystrophy. (DMD). It is important to understanding the similarities and differences in pharmacokinetics (PKs) among antisense oligonucleotises (AOs) classes. For the class 2'-O-methyl phosphorothioate (2'-O-methyl PS) most pharmacokinetics data available, are derived from development of exon skipping therapy for DMD. And, 2'-O-methyl PS also contains most of the properties for being prominent antisense oligonucleotide (AOs) for exon skipping therapy in DMD.

gladiator answered 4 months ago

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

The nurse is caring for a child that has been diagnosed with Duchenne Muscular Dystrophy (DMD). The nurse understands DMD: Group of answer choices Is a progressive muscle weakness disorder. Has an increase in dystrophin levels. May cause respiratory muscle weakness. Will cause elevated creatinine kinase levels. Spasticity of muscles.

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

Fill in the blank. Duchenne muscular dystrophy is caused by an X-linked recessive mutation in the DMD gene, which encodes the protein dystrophin. A female without the disease mates with a male with Duchenne muscular dystrophy; they have two sons and two daughters. The first son has the disease as does their first daughter. Based on this, the probability that the second son will have the disease is________________ The probability that the second daughter will be a carrier is____________

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?

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

Outline a workflow for the diagnosis of Duchenne Muscular Dystrophy. Include what patient details and material may be assessed and what would be detected/observed at each point of the workflow.

A patient with Duchenne muscular dystrophy presents with fibrosis, where some of the contractile muscle fibers...

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...

Fill-ins. Type the one BEST word or short phrase into the answer box. Duchenne muscular dystrophy...

Fill-ins. Type the one BEST word or short phrase into the answer box. Duchenne muscular dystrophy is caused by an X-linked recessive mutation in the DMD gene, which encodes the protein dystrophin. A female without the disease mates with a male with Duchenne muscular dystrophy; they have two sons and two daughters. The first son has the disease as does their first daughter. Based on this, the probability that the second son will have the disease is ____________. The probability...

QUESTION 7. Assay question Duchenne muscular dystrophy is one of the sex-inherited disease. Write a report...

QUESTION 7. Assay question Duchenne muscular dystrophy is one of the sex-inherited disease. Write a report about the mutation that caused the disease, symptoms, genetics therapies, include a minimum of one human pedigree that show the disease. Note: 1. A minimum of THREE scientific papers are encouraged to be used 2. Include a reference list with proper citation (Harvard style). 3. The report should not exceed 100 words

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

3. Why is carrier status for Duchenne muscular dystrophy (DMD) usually not transmitted from affected fathers to daughters, even though this disorder has an X-linked transmission pattern 4. Why is there such a high rate of spontaneous mutation among the genes responsible for classic hemophilia, Duchenne muscular dystrophy, and achondroplasia?

1. Duchenne muscular dystrophy (DMD) is caused by a relatively rare X-linked recessive allele. It causes...

1. Duchenne muscular dystrophy (DMD) is caused by a relatively rare X-linked recessive allele. It causes progressive muscular wasting, and usually leads to death before age 20. What is the probability that the first son of a woman (who does not have the disease but whose father is affected) will be affected? 2. Hemophilia in humans is inherited as an X linked recessive trait. A woman whose father is hemophiliac marries a man with normal clotting ability. What is the...

Many early video artists used ___. 1. still photographs 2. magnetic tape that records both image...

Many early video artists used ___. 1. still photographs 2. magnetic tape that records both image and sound 3. motion capture technology 4. computer programming

Question