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GENE THERAPY_ Powered By Docstoc



   1. What is gene therapy?

   2. What is a mutation?

   3. What happens most times with a mutation?

   4. What is a gene?

   5. Mutations cause the ______ encoded by that gene to malfunction.

   6. When a protein malfunctions, cells that rely on that protein's function can't
      behave normally, causing problems for whole tissues or organs. Medical
      conditions related to gene mutations are called genetic ________ .

   7. What are the options to fix a flawed gene ?




   8. If it is successful, ___________ provides a way to fix a problem at its
      source. Adding a corrected copy of the _________ may help the affected
      cells, tissues and organs work properly. Gene therapy differs from
      traditional drug based approaches, which may treat the problem, but
      which do not repair the underlying __________ flaw.

Read the Gene Therapy Case Study Section 1:

   1.   What does “CF” stand for?
   2.   What organs do CF affect.

   3.   What symptoms does CF cause?

   4.   What is the prognosis for CF if left untreated?

   5.   Which gene causes CF?

   6.   What does CFTR do in normal cells?

   7.   Specifically what happens when the CFTR gene is mutated in a cell?


   1. Gene therapy could potentially treat certain disorders _______ by
      repairing the underlying _______ flaws.

   2. For any candidate disorder, you need to answer the following 5 questions:





Read the Gene Therapy Case Study Section 2:

   1. Does the condition result from mutations in one or more genes?

   2. What type of disorder is CF?

   3. Is CF a single gene disorder?

   4. What do you know about the biology of CF?
   5. Will adding a normal copy of CFTR fix the problem with CF?

   6. What could be used to treat CFTR ?


   1. In Choosing Targets for Gene Therapy, we saw that cystic fibrosis is a
      good candidate for gene therapy. This is because:

      We know which _______

      We have ______________ gene available.

      We understand ___________________,

      We can predict that adding __________ to the cells will restore a needed

   2. What are the FOUR of the hallmarks of successful gene delivery? Briefly
      explain each






   1. Scientists refer to these DNA delivery "vehicles" as ___________ ?

   2. Click the vector tool box and list the 6 vectors and the type of vector each
      of the 6 are.






  3. Part of the challenge in gene therapy is choosing the ________ for
     treating the disorder.

  4. List 3 advantages of viral vectors:




  5. List 2 drawbacks of viral vectors:



  6. Non-viral vectors are known as _______

  7. What is a plamid?

Read the Gene Therapy Case Study Section 3:

      What is the best vector to treat CF ?

   1. Taking gene therapy from the laboratory to the clinic involves many steps.
      Before trying a therapy on human patients, researchers must (list 4 )

   2. Let's look at a real-life example: development of a gene therapy treatment
      for a disorder called Adenosine Deaminase (ADA) deficiency. List the 8
      steps needed to follow in order to advance gene therapy treatment for









   3. What are the three primary sources for funding these 8 steps?




   4. How many people are in a phase 1 trail?
   5. How many people are in a phase 2 trail?

   6. How many people are in a phase 3 trail?

   7. As of the date of this article: Where the trials to test a Gene Therapy for
      ADA successful?

Read the Gene Therapy Case Study Section 4:

         What two parts of the vector were removed to ensure the safety of the
         gene therapy study?



   1. How long has gene therapy been around?

   2. How hard is it to introduce new genes into existing cells?

   3. How many cells must gene therapy be introduced to (at minimum) to be

   4. What is the germline?

   5. Why is it important?

   6. Gene vectors must also be able to escape what bodily function?

   7. Why did Jessie Gelsinger die?
   8. The best gene therapy is the one that lasts. For this to happen, the newly
      introduced gene must become a permanent part of each cell's ______.

   9. What is a genome?

   10. But what happens if the gene stitches itself into an inappropriate location,
       disrupting another gene. As an example what disease did children with
       Severe Combined Immune Deficiency (SCID) later develop because of
       their gene therapy experience.

Read the Gene Therapy Case Study Section 5:

   1. In what year did gene therapy begin to treat CF?

   2. Which Vector was used first to treat CF?

   3. Why did it not work?

   4. What did researchers try to use in 1995?

   5. Why did it not work?

   6. What did researches try to use in 1998?

   7. Why was it successful?

   8. Why was it not a complete success?


Answer the following using at least 8 sentences each.

   1. When should gene therapy be used? Should it be used to treat critically
      ill patients? Should it be used to treat babies and children?
2. What effect would gene therapy have on future generations if
   germline (reproductive) cells were genetically altered? How might this
   alteration affect human variation?

3. Who should decide what are "good" or "bad" uses of genetic
   modifications? How do you define "normal" with regard to human

4. What if we could alter human traits not associated with disease?
   Would it be okay to use gene therapy to improve or enhance a person's
   genetic profile?

5. Who will have access to gene therapy, treatments and long-term
   follow-ups? Will gene therapy and genetic enhancements create an
   advantage for those who can afford it?
Complete the following chart:

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