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Gene Therapy

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Gene Therapy AP Biology Unit 2 What is Gene Therapy? A way to treat or cure diseases by inserting the correct DNA into the cell. Most promising for diseases ... – PowerPoint PPT presentation

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Title: Gene Therapy


1
Gene Therapy
AP Biology Unit 2
2
What is Gene Therapy?
  • A way to treat or cure diseases by inserting the
    correct DNA into the cell.
  • Most promising for diseases caused by a single
    gene defect (ex. hemophilia)
  • Still in the experimental stages
  • Clinical trials have been done on humans

3
The Big Picture
  • Image taken without permission from
    http//www.fda.gov/fdac/features/2000/gene.html

4
Ex vivo
  • Means it occurs outside of the body
  • Steps
  • Defective cells are taken out of the patient
  • Add functional gene into defective cells
  • Return these cells to the patients body
  • Has been used to treat hemophilia, and adenosine
    deaminase deficiencies (leads to immune system
    problems)

5
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6
In vivo
  • Means it occurs in the patients body
  • Insert the genes directly into the patients
    cells
  • Ex. using an aerosol spray containing functional
    genes that are are mutated in cancer cells to
    treat lung cancer.
  • Still in the early stages of experimentation

7
Challenges
  • Find appropriate vectors
  • currently recombinant viruses and plasmids are
    being tested
  • Make sure vectors can be taken up by human cells
    properly
  • Ensure that new gene is inserted into human
    genome properly and proteins are expressed
    correctly
  • Could be very problematic if it inserts itself in
    the wrong place and disrupts transcription of a
    crucial protein

8
More challenges
  • How to make sure the functional gene gets to all
    the cells that need it
  • How to sustain the cells that got the functional
    gene cells die after a while!
  • How to control how the immune system responds to
    the vector
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