GEN #19 Gene Therapy II

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  1. a _______ mutation is the easiest to treat
    loss of function
  2. The ______ the size of DNA to be introduced the more difficult in general.
    the larger
  3. ________ levels vary in response to metabolic or physiological mediators
    gene expression
  4. target tissues that are the most difficult to target are _______
    Internal organs
  5. ADA is an enzyme in the
    purine salvage pathway
  6. ADA stands for
    Adenosine Deaminase
  7. ________ are particularly sensitive to decreased ADA activity because they rely heavily on the ________________.
    • T-lymphocytes
    • salvage pathway forpurines
  8. ADA is characterized by a __________ immunodeficiency syndrome
    combined B & T-cell
  9. CFTR stand for
    cystic fibrosis transmembrane conductance regulator
  10. The most common CF defects is ______
    ΔF508 mutation
  11. The CFTR gene os located on the
    7q31.2 chromosome
  12. AAV stands for
    Adeno-associated virus
  13. The first gene therapy trial used an adenoviral vector in which ________ and _______ had been deleted.
    E1 & E3
  14. The three non-viral vector systems are
    • Cationic liposomes
    • DNA-polymer conjugates
    • Naked DNA
  15. Wiskott-Aldrich Syndrome is an
    X-linked recessive disease
  16. WAS is characterized by
    • Eczema
    • Thrombocytopenia
    • Immune deficiency & development of autoimmune diseases
  17. WAS gene codes for protein that regulates _________ in immune cells.
    actin polymerization
  18. WAS stands for
    Wiskott-Aldrich Syndrome
  19. Ex vivo approach to gene therapy for WAS using a retroviral
    vector obtained ___________ by ___________
    • CD34+ [hematopoietic stem cells]
    • leukaphoresis
  20. WAS-expressing retroviral vector composed of the ________ backbone virus pseudotyped with the gibbon ape ________ virus envelop protein.
    • CMMP
    • leukemia
  21. In the PD clinical trial the AAV vector contained the gene for ___________ which is required for __________ biosynthesis
    • glutamic acid decarboxylase (GAD)
    • GABA
  22. The vector for PD patients was injected into the ________ region of the brain
Card Set
GEN #19 Gene Therapy II
GEN #19 Gene Therapy II
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