Term
| in theory what can gene therapy treat |
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Definition
| infections that do not have a cure |
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Term
| in one sentence, tell how gene therapy works |
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Definition
| transfer specific genetic information (usually DNA) into patient to treat disease |
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Term
| potentially in the future, what could gene therapy treat |
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Definition
| genetic diseases (monogeme) and cancer |
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Term
| what is somatic gene therapy |
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Definition
| gene delivered only to somatic cells, not effect to germline. |
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Term
| what is the type of gene therapy used in people today |
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Definition
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Term
| what is germline gene therapy |
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Definition
| affects somatic and germline or only germline cells |
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Term
| what makes germline gene therapy different from somatic |
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Definition
| affects germline, perminate, heritable, ethical issues |
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Term
| what is germline gene therapy currently used for |
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Definition
| inducing human diseases in lab animals to use them in experiments, nothing in humans |
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Term
| to have successful gene therapy what do you need |
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Definition
| efficient gene delivery system, theraputic genes |
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Term
| what is the function of a gene delivery system |
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Definition
| get the gene to the right cell, the the gene inside the cell, express the gene |
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Term
| what gene delivery system is most common in gene therapy in humans |
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Definition
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Term
| why do we use viral gene delivery systems |
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Definition
| viruses already have the machinery to get in a cell with genetic material and integrate it into human DNA |
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Term
| how can we use a viral gene delivery system when it is well... a virus |
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Definition
| the viral pathogen genes are removed / blocked but the genes used to get in the cell and integrate DNA are not. despite this it could still cause problems |
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Term
| what is transient expression |
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Definition
| gene inserted through gene therapy is not replicated through normal cell gene replication |
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Term
| if a gene has transient expression, how can you make gene therapy successful anyways |
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Definition
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Term
| what are some issues that can nix your gene therapy efforts even though you have a good gene delivery system |
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Definition
| new gene knocks out function of another gene when inserted, difficult to control expression, immune system may reject the cells with the gene (apoptosis) |
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Term
| what are the three most common vector systems used in gene therapy |
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Definition
| retroviral, adenovirus, non-viral |
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Term
| what are the benifits of using retrovirus vectors in gene therapy |
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Definition
| high efficiency transduction into cells infecting a lot of them, inserts into the chrosomosone (not transient) |
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Term
| what are the negatives of using retrovirus vectors in gene therapy |
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Definition
| need to insert into dividing cell, mutations due to insertion, size limits of gene, risk of infection |
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Term
| what are the benifits of using adenovirus vector during gene therapy |
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Definition
| good transduction into cells and affects lots of cells, don't have to wait for dividing cell, broad range of target cells, does not insert into chromosome |
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Term
| what are the negatives of using adenovirus vector in gene therapy |
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Definition
| transient expression, immune rejection possible, become resistant over time, risk of infection |
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Term
| what are the benifits of using a non-viral vector in gene therapy |
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Definition
| no infection risk, decreased immune response, synthetic, no size limitation |
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Term
| what are the negatives of using non-viral vector in gene therapy |
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Definition
| low efficiency getting into and infecting cells, limited target cell possibilities, transient expression |
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Term
| what is gene augmentation therapy |
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Definition
| give extra copy of a normal gene |
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Term
| what does gene augmentation therapy treat |
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Definition
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Term
| explain targeted killing of specific cells as a gene therapy |
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Definition
| toxic gene is delivered or expressed only in bad cells (like cancer cells) |
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Term
| explain the gene therapy that uses target inhibition of gene expression |
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Definition
| at level of DNA, RNA, or protein block transcription, translation, or degrate proteins |
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Term
| what is targeted inhibition gene therapy used in |
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Definition
| cancer gene therapy, some autosomal dominent diseases (huntingtons) |
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Term
| explain how to do targeted gene mutation correction gene therapy in DNA |
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Definition
1. target DNA
2. provide copy of normal sequence
3. replace mutated sequence piece (homologous recombination) |
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Term
| explain how to do targeted gene mutation correction gene therapy in RNA |
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Definition
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Term
| what may be the only acceptable germline gene therapy |
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Definition
| targeted gene mutation correction |
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Term
| what is defficient in severe combined immunodeficiency disease, how ofted |
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Definition
| adenosine deaminase 10% of cases |
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Term
| what type of inheritance is severe combined immunodeficiency disease |
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Definition
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Term
| what process is affected in severe combined immunodeficiency disease |
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Definition
| lymphocyte nucleotide metabolism do DNA replication is inhibited |
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Term
| what is lacking in someone with severe combined immunodeficiency disease |
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Definition
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Term
| what is the symptom of severe combined immunodeficiency disease |
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Definition
| suspectability to infection, must stay in a sterile enivornment |
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Term
| what is the first example of successful gene therapy |
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Definition
| severe combined immunodeficiency disease |
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Term
| why did severe combined immunodeficiency disease not work in gene therapy |
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Definition
| tried using stem cells instead but people developed lukemia |
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Term
| list the steps of doing gene therapy on someone with severe combined immunodeficiency disease |
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Definition
1. clone normal adenosine deaminase
2. put it in vector
3. isolate patient lymphocytes
4. transfect the lymphocyte with retrovirus vector
5. reverse transcriptase RNA to DNA
6. insert DNA into lymphocyte
7. inject lymphocyte into patient
8. expression of gene transforms lymphocyte regaining normal immune function |
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Term
| what are the steps in cloning a mammal |
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Definition
1. remove nucleus from egg cell
2. remove nucleus from animal you want to clone
3. inject nucleus into empty egg cell |
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Term
| what are the issues with cloning mammals |
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Definition
| mitochondrial DNA is not cloned, shortened temomeres may lead to shortened life span |
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