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  #1  
Old 09-19-2012
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Star Genetics HY

A patient suffers from adenosine deaminase (ADA) deficiency, an autosomal recessive immune deficiency in which bone marrow lymphoblasts cannot replicate to generate immunocompetent lymphocytes. The treatment option that would permanently cure the patient is:

a. Germ-line gene therapy to replace one ADA gene copy
b. Germ-line gene therapy to replace both ADA gene copies
c. Somatic cell gene therapy to replace one ADA gene copy in circulating lymphocytes
d. Somatic cell gene therapy to replace both ADA gene copies in circulating lymphocytes
e. Somatic cell gene therapy to replace one ADA gene copy in bone marrow lymphoblasts

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  #2  
Old 09-19-2012
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SCID...and the answer is WTF !!!
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Old 09-19-2012
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Default WTF qs

yeah buddy "too wtf", but it has a very reasonable answer
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Old 09-19-2012
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Default my answer :)

very nice question!

I'll go with e. Somatic cell gene therapy to replace one ADA gene copy in bone marrow lymphoblasts


I excluded a) and b) as it's not done in humans - ethics issues.
Replacing 2 genes would be perfect, imho. But the disease is recessive so 1 replacement should do. And it makes much more sense to replace defective gene in bone marrow progenitor cells rather than the lymphocytes that are already circulating.
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  #5  
Old 09-19-2012
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Thumbs Up Correct answer is e

wowww it`s a too fast "KNOCK OUT" jejeje, yup Casandra your answer is correct

Here is the explanation:


Gene therapy refers to a group of techniques by which gene structure or expression is altered to ameliorate a disease. Because of ethical and practical difficulties, germ-line therapy involving alterations of genes in primordial germ cells is not being explored in humans. Although germ-line genetic engineering is being performed in animals with the goals of improved breeding or agricultural yield, it alters the characteristics of offspring rather than the treated individuals. Somatic cell gene therapy is targeted to an affected tissue or group of tissues in the individual, and is most effective if stem cells such as bone marrow can be treated. Somatic cell gene therapy offers the hope of replacing damaged tissue without the rejection problems of transplantation. For autosomal recessive disorders, only one of the two defective alleles must be replaced or supplemented.
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  #6  
Old 09-20-2012
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Quote:
Originally Posted by Fuser00 View Post
A patient suffers from adenosine deaminase (ADA) deficiency, an autosomal recessive immune deficiency in which bone marrow lymphoblasts cannot replicate to generate immunocompetent lymphocytes. The treatment option that would permanently cure the patient is:

a. Germ-line gene therapy to replace one ADA gene copy
b. Germ-line gene therapy to replace both ADA gene copies
c. Somatic cell gene therapy to replace one ADA gene copy in circulating lymphocytes
d. Somatic cell gene therapy to replace both ADA gene copies in circulating lymphocytes
e. Somatic cell gene therapy to replace one ADA gene copy in bone marrow lymphoblasts

Would go with

e. Somatic cell gene therapy to replace one ADA gene copy in bone marrow lymphoblasts

As its SCID or bubble boy syndrome. Which is treated with some bone marrow transplant stuff( once saw in discovery). Hahha
I know pretty lame but truely telling my reasoning guys..hehehe

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  #7  
Old 09-20-2012
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Thanks for the question! Jeez, these scare me!!

PS. And you guys are truly hilarious!! Haha!!
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