GEN #18 Gene Therapy I unfinished

Card Set Information

GEN #18 Gene Therapy I unfinished
2012-03-26 11:38:50

GEN #18 Gene Therapy I
Show Answers:

  1. The manipulation of an individuals genomic information to produce a therapeutic outcome is _______
    gene therapy
  2. A liver disease that cause high ammonia levels and is abbreviated OTC is
    ornithine transcarbamylase deficiency
  3. ADA deficiency causes
    severe combined immunodeficiency syndrome
  4. Ways to treat acquired diseases are
    • Gene augmentation therapy
    • Eliminate selected cell populations
    • Correct an acquired gene mutation
  5. Gene augmentation therapy refers to an acquired ______ function
    loss of gene
  6. Generally the introduction of_______will overcome a loss of gene defect
    a single gene
  7. ___________ removes a specific cell population from the body
    Elimination of Selected Cells
  8. Inserting a gene to code for a cellular toxin is an example of ________
    Elimination of Selected Cells
  9. Direct killing of diseased cells can be done by inserting a __________ or a ___________
    • toxin gene
    • prodrug gene
  10. Prodrug activation therapy requires viral _________ to be transfected into cells
    thymidine kinase
  11. ganciclovir is ___________ by the viral TK
  12. _________ finish adding phosphate and ganciclovir blocks cell division.
    host cells
  13. ganciclovir monophosphate enters adjacent cells through ______ formed by ________
    • gap junctions
    • connexon proteins
  14. ganciclovir monophosphate is ________ in the adjacent cell and kills that cell as well
  15. _________ substitutes a wild-type gene in place of a gene that has been altered due to chemical, viral, or physical injury
    Correction of a Gene Mutation
  16. Correction of a Gene Mutation uses __________ to do replacement
    homologous recombination
  17. Targeted Gene Editing disrupts a single gene using a _______ approach
    zinc finger nuclease
  18. zinc finger nuclease is an engineered fusion protein containing
    • DNA cleavage domain of the restriction endonuclease Fokl
    • Zf DNA binding domain
  19. Fokl needs to ______for activity using two ________ proteins
    • dimerize
    • nonpalindromic
  20. If DNA binding domain contains ________ covering ___ zinc finger domains, will provide unique specificity of binding.
    • 18 bp
    • 3
  21. 2 most popular viral systems are
    • Retroviral vectors
    • Adenoviral vectors