BIOTEC E3 C10

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shockwave
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BIOTEC E3 C10
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2011-11-30 22:34:08
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BIOTEC E3 C10
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  1. Gene Therapy. WHAT CELLS ?
    Somatic cell treatment
  2. GENETIC DISORDERS. NAME 4 CATAGORIES
    • Single-gene changes..SICKLE CELL
    • Multigene disorders..CANCER/HEART/DIABETES
    • Mitochondrial disorders...MITO DNA ISSUES
    • Chromosome abnormalities...DOWN SYNDROME
  3. Several factors must be considered for a disease to be a candidate for gene therapy:
    • -single gene causing a genetic disorder
    • -identified The normal and mutated genes must be well-studied
    • -disease caused by the mutation
    • must be well-understood
    • -gene delivery method must be available
    • -produces an immune response?
    • -delivered to the correct cells
    • -integrated into the host chromosome
  4. For a disease to be a candidate for gene therapy...MAJOR ITEM
    gene must be turned on (transcription) at the right time, and is also regulated properly
  5. There are two major strategies for the insertion of genes into cells.
    Ex vivo and In Vivo
  6. Ex vivo gene therapy BIGEST ISSUE
    The transplantation of the cells is the biggest technical problem
  7. EX VIVO ADVANTAGE OVER IN VIVO
    advantage over in vivo gene therapy is that rejection does not occur if the person’s own cells are used
  8. EN VIVO....4 STEPS
    • Cells are removed from the body
    • The gene of interest is inserted into them
    • The cells are cultured for reproduction
    • The cells are returned to the body
  9. IN VIVO...2 ISSUES
    The transferred gene is unstable and the product produced temporarily

    The methods are not as controlled as ex vivo gene therapy because cells are not removed from the body
  10. Retrovirus:
    RNA viruses that only infect ------ human cells

    DNA of interest can only be up to ----- kb in size
    • dividing
    • 8
  11. NAME 6 WAYS TO TRANSFER DNA INTO CELLS
    • RETROVIRUS
    • ADENOVIRUS
    • ADENO-ASSOCIATED VIRUS
    • HERPIES SIMPLES VIRUS
    • LIPOSOMES
    • NAKED DNA
  12. WHAT VIRUS CAN INVECT DIVIDING AND NONDIVIDING CELLS?
    ADENOVIRUS AND ADENO-ASSOCIATED
  13. Can engineer proteins on the virus surface to target specific cells
    VIRUS TYPE?
    ADENOVIRUS
  14. ADENOVIRUS:

    DNA of interest can only be up to ----- kb in size
    7.4 KB AND PROTEIN WILL BE HIGHLY EXPRESSED
  15. Does not integrate into the genome, so there is low risk of mutation...WHAT VIRUS?
    ADENOVIRUS
  16. 95% of the time DNA integrates into a region in chromosome 19...WHAT VIRUS
    ADENO-ASSOCIATED
  17. DNA of interest can be up to 5 kb..WHAT VIRUS ?
    ADENO-ASSOCIATED
  18. Need a helper virus to infect host cells
    WHAT VIRUS TYPE?
    ADENO-ASSOCIATED
  19. DEFINE LIPOSOMES
    membrane-bound spheres that contain the DNA of interest...DELIEVERY METHOD
  20. 1ST GENE THERAPY LANDMARK
    • 1990 4YR OLD GIRL WITH SCID.
    • treated with T-cells with adenosine deaminase gene in stem cells that permanently corrected the genetic disorder
    • The therapy had to be repeated because the enzyme would be produced for only a few
    • months and then stopped
    • More than 50% of the T cells in the girl contained the corrected gene
    • 2003 her body still actively produces ADA
  21. WHAT THE HELL IS SCID?
    • Defective adenosine deaminase (ADA) gene
    • = immune malfunction

    Allows for build-up of toxic metabolic by-product
  22. 2ND GENE THERAPHY CASE
    • 11-year-old girl
    • Immune system developed a reaction against the virus
    • Only 0.1% to 1.0% of her cells produced ADA
  23. Therapies aimed at lowering LDL by transfecting liver cells with the gene for the LDL receptor using
    ---- & ------- vectors
    retroviral and adenoviral.

    IN SOME CASES....EX VIVO...BAD RECIEPTOR GENES.
  24. Transfected ----------- have not been inserted into the liver with much success
    hepatocytes (liver cells)

    Only about 10% of the cells incorporating into the liver.
  25. CF transmembrane transconductance regulator (CFTR) in the plasma membrane of a cell .....WHAT ORGANS?
    • airways,
    • intestines,
    • pancreas
  26. NAME TWO TREATMENTS FOR CFTR
    small amount of molecules need to be produced per cell

    Spraying adenoviruses with the CFTR gene into the nose has had only temporary treatment

    spraying a DNA-liposome aerosol into the nose, but it may not affect distant organs
  27. CF transmembrane transconductance regulator (CFTR) in the plasma membrane of a cell..HOW DOES IT WORK?
  28. NAME ORGANS USED IN GENE THERAPY
    • LIVER
    • LUNG
    • hematopoietic (BLOOD)
    • Circulated Gene Products (HORMONE IN CAPSULES)
  29. DEFINE Hematopoietic diseases
    Cells of circulatory system are produced by pluripotent, undifferentiated hematopoietic stem cells in bone marrow

    Erythrocytes, leukocytes and platelets are made in response to erythropoietin and colony stimulating factor

    Any stem cells that are transfected would give function for life of the individual
  30. WHAT ARE Circulated Gene Products?
    • Encapsulated recombinant cells
    • -Uses include timed production of human growth hormone inside the capsule
  31. Is Gene Therapy Safe?
    NAME 2 CASES
    Gelsinger An 18-year-old with ornithine transcarbamylase (OTC) deficiency. vectors used formed clotts--> death.

    • SCID/ Leukemia:
    • Retrovirus used inserted the DNA next to an oncogene (a gene that can lead to cancer) in a white blood cell-->uncontrollable growth
  32. T OR F ?
    No one has completely been cured by gene therapy
    TRUE
  33. The first commercially licensed gene therapy.
    • In October 2003 in China, under the name Gendicine,
    • after 5 years of clinical trials.
    • Treats head and neck squamous cell carcinoma.
    • ONLY SIDE EFFECT...LOW FEVER
  34. The first commercially licensed gene therapy...WHAT VECTOR?
    • An adenovirus vector containing the
    • p53 tumor suppressor gene; Can be used along with chemotherapy and radiation therapy to increase effectiveness.
  35. DEFINE dominant negative gene
    • Mutated gene can prevent a normal protein from functioning properly.
    • Cannot be corrected by simply inserting the correct gene
  36. DEFINE SMaRT
    • Spliceosome Mediated RNA Trans-splicing.
    • Instead of correcting the gene, the region of mRNA that is affected is repaired
  37. HOW DOES SMART WORK?
    RNA strand pairs with intron (by base pairing) next to mutated region (exon) into cells

    modified to contain the correct exon and a small region that binds to the neighboring intron.

    strand binds to the intron, the duplex (section of double-stranded RNA) causes the spliceosome to cut and remove the intron and the defective exon from the mRNA.

    exons are joined, with the corrected exon ligated into the mRNA, thereby generating a functional, mature mRNA and protein
  38. DEFINE Triplex-Helix-Forming Oligonucleotide Therapy
    single-stranded string of nucleotides, 15-21 bases long binds to the groove between the double strands of DNA where the mutated gene is used---TRIPLE HELIX FORMS..BLOCKS TRANSCRIPTION..NO mRNA.CORRECT GENE INTO CELL PRODUCE PROTEIN.
  39. WHAT IS THIS?
    DEFINE Triplex-Helix-Forming Oligonucleotide Therapy
  40. Spliceosome Mediated RNA Trans-splicing (SMaRT)
  41. DEFINE Antisense Therapy
    Targets the mRNA of a mutated gene so that it cannot be translated into protein

    Is complimentary to the mRNA that is used to code for the protein, called the “sense” mRNA
  42. Antisense Therapy The steps of the method are:
    • “antisnense” RNA is introduced into cells that is complimentary to the mRNA that is used to
    • code for the protein, called the “sense” mRNA

    The antisense RNA binds to the sense mRNA strands synthesized by the cell during transcription

    duplex RNA is blocked from translation into a protein

    Eliminating or dramatically reducing the production of a mutated protein
  43. Antisense Therapy

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