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Discussion 10

Discussion 10

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Gene Therapy: Many great drugs assist to alleviate the symptoms of diseases and even to help

people live fairly normal lives despite their underlying disease states. However, some diseases

are not well-controlled with drugs, including cancer, Parkinson disease, hemophilia, and others.

For such diseases, researchers have been searching for a possible cure, and believe that they may

have a way to do so using gene therapy. The idea behind gene therapy treatments is that, by

injecting DNA for the normal protein product into the patient’s cells, we can change the gene

product in those cells so that the patient’s symptoms are eliminated. Ultimately, the goal is to

inject DNA molecules that can completely get rid of the mutant gene in the cell and replace it

with functioning, normal copies of the genes, essentially CURING disease.

For our Discussion topic this week,

• Read the Special Topics section in the back of your textbook on the "Gene Therapy," found on

page 519-530.

For our discussion this week, include the following in your initial post:

• What you knew about gene therapy before reading this section

I knew that gene therapy is a newer form of treatment which revolutionizes the way some

of the most deadly diseases, who are otherwise not considered curable can be cured.

• What questions you had while reading this section

While reading this section, I had the question that how can a normal DNA injected into a

cell having abnormal DNA replace the entire abnormally arranged DNA in the mutant

genes. I mean to say how this is actually carried out in the patients? Is it a very controlled

or monitored process or what?

• One cool thing you learned about gene therapy

One cool thing I have learned about gene therapy is that it can cure cancers.

• Think of a disease that would be a good target for gene therapy. Explain why it would be

a good target (why is gene therapy going to be the only way to effectively target the

disease?).

Duchene Muscular Dystrophy (DMD) in children seems to be a good target for gene

therapy. Since, as of now, no drug or medication can reverse the dystrophic changes

occurring in muscles in DMD as of now and since it occurs due to abnormalities in genes,

I think gene therapy is the only way to treat it.

• Answers to questions 1-3 of the “Discussion Questions” section on page 530

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Gene Therapy: Many great drugs assist to alleviate the symptoms of diseases and even to help people live fairly normal lives despite their underlying disease states. However, some diseases are not well-controlled with drugs, including cancer, Parkinson disease, hemophilia, and others. For such diseases, researchers have been searching for a possible cure, and believe that they may have a way to do so using gene therapy. The idea behind gene therapy treatments is that, by injecting DNA for the normal protein product into the patient’s cells, we can change the gene product in those cells so th ...
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