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39 Cards in this Set
- Front
- Back
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1st gene therapy treatment treated what disease?
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SCID caused by adenosinedeaminase deficiency (ADA deficiency )
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The main reason ADA gene therapy worked
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Corrected cells had a growth advantage in vivo
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Some diseases that are candidates for treatment by gene therapy
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SCID
Leber's congenital amaurosis Restenosis Hemophillia B and A Parkinsons Stroke Storage Diseases (mutated lysosomal enzymes) |
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Restenosis uses what kind of vector?
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an HVJ (Sendai) liposome gene delivery system
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Hemophilla B patients are deficient in?
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Factor IX
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Requirements for Gene Therapy of Hemophilia B
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Efficent gene transduction
Long term persistence of transduced gene Appropriate expression of functional factor IX Factor IX expression at therapeutic levels |
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How do parkinson's treatments with gene therapy work?
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1. Boost levels of tyrosine hydroxylase to increase dopamine production in remaining neurons
2. transplant fetal neurons infected with bcl-2 gene which prevents apoptosis |
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Methods used to prevent stroke's neuronal death effects
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1. introduce the bcl-2 gene to prevent cell death
2. introduce a gene that encodes for a protein that elevates glucose uptake |
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What is sly syndrome?
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A storage disease in which fatty acids accumulate in the brain due to B-glucuronidase gene mutation.
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Name some gene therapy approaches to cancer.
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1. Introduce immune system stimulating genes like IL2 and B7 into cancer cells. Targeting tumors is an issue
2. introduce antisense RNA to block bad genes like oncogenes. Targeting is an issue. 3. Introduce tumor suppressor genes 4. Introduce thymidine kinase gene which sensitizes cells to gancyclovir. 5. Introduce drug resistance genes into bone marrow to allow stronger doses of chemotherapy. |
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Name 4 functions of compliment
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Lyse microorganisms
Coat pathogen, making it easier to phagocytosize Release substance like histamine to increase inflammatory response Attract lymphocytes to infection sites |
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When the antigen antibody complex forms, what 4 things can occur?
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Activation of compliment
Inactivation of pathogen Activation of phagocytes that ingest pathogens Binding to the Fc receptors of phagocytes |
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advantages of using Adenoviruses
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+Can integrate large exogenous genes
+efficient gene transfer +can infect non-dividing cells |
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disadvantages of using Adenoviruses
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+very immunogenic
+since they don't integrate into host's genome, gene expression is transient |
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Advantages of AAVs
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+not as immunogenic as AV
+site specific integration |
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Disadvantages of using AAV
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-limited packaging capacity
-stable helper cell lines not available -need a helper virus to replicate -less efficient integation -deletions/rearrangements occur frequently -no AAV vectors have been show to integrate into C19 |
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What viral vector can be used to target the respiratory tract?
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Papovirus
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name two advantages of using HSV as a vector?
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It naturally encodes thymidine kinase (a target for gancyclovir) and targets the nervous system.
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Sinbis virus can be made at high titers and have a short expression in host cells because they do not integrate into host genome, T/F?
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True!
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Name the 2 major kinds of gene therapy
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Germline and somatic
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Has germline therapy been used in humans yet?
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No
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Ex Vivo transfers are mostly used with what kind of cells?
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Blood and bone marrow cells, possibly skin and liver in the future.
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Advantages of Ex Vivo systems
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Efficient gene transfer
Vectors dont need to be tissue specific Target cells can be easily manipulated |
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Advantages of non-viral vectors
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+cheap, easy to use
+non-immunogenic +can incorporate large pieces of DNA |
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what gene is produced in transgenic sheep milk?
and what is the method used to make it? |
alpha 1- antitrypsin gene
A1AT protective function in lungs Pronuclear injection |
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What is a more regulated way to introduce a gene into somatic cells of a transgenic animal?
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Gene targeted nuclear transfer method
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Two examples of drugs developed by pharming that have advanced to clinical trials.
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1. Antithrombin III (AT III)
2. Alpha glucosidase (GAA) |
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What are two methods for producing transgenic chickens?
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1. transfect embryos with viral vector containing the transgene
2. insert transgene into rooster sperm |
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What are major obstacles to porcine xenotransplantation?
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1. Production of antibodies against 1,3 Gal
2. loss of clotting factors after transplant 3. excessive amounts of vascular cell adhesion molecules generates immune response Solutions |
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How is Cre tissue specific?
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It is expressed with a tissue specific promoter.
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For the knockout of the NMDA receptor, which subunit was knocked out of what region?
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NR1 subunit of the CA1 region of the hippocampus
This area appears to be important for memory |
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What subunit is found in young individuals whose receptors stay open for longer allowing for more learning?
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NR2B
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What are the four transgenes used to develop iPS
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Sox 2: necessary for embryonic development and for prevention ES cell differentiation
Oct 3/4: implicated in maintenance of pluripotency C-Myc: important for proliferation but also an onco gene Klf4: its overexpression inhibits differentiation |
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What is iPS?
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Induced Pluripotent Stem Cells
the generation of stem cells from adult skin cells (fibroblasts) by introducing four genes |
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Advantages to using iPS
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no need for killing babies (embryos)
avoid rejection problems; genetically matched |
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Challenges in the use of iPS
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Requires use of retrovirus, which can lead to cancer.
Requires the expression of genes involved in cancer (c-Myc) Takes time to generate cells |
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Advantage of nuclear transplant stem cells
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-avoid tissue rejection problems
-can use genetic engineering |
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_______ Cysts are evidence that spontaneous activation occurs in nature.
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Dermoid
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Egg activation without sperm is called
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parthenogenesis. Eggs are refered to as parthenotes. These benign teratomas can lead to derivations of stem cell lines.
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