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14 Cards in this Set
- Front
- Back
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Gene therapy
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replaces malfunctioning gene, alleviates symptoms
Controversial |
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Eugenics
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breed better humans by encouraging reproduction of ppl with "good" genes and discouraging reproduction of ppl with "bad" genes
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therapeutic vs. enhancement
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therapeutic: attemt to cure a disease
enhancement: attempt to chose a child's tratis (stregnth, intelligence) |
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SCID
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Immune system is nonfunctional (ADA deficiency, Xlinked)
only human disease to be cured by gene therapy |
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Xlinked SCIDS
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14 children treated w/ gene therapy
3 developed leukemia (insertion of retroviral vector near proto-oncogene LMO2 promoter led to uncontrolled proliferation of mature T cells) |
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OTC trial
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Ornithine transcarbamylase deficiency: lack enzyme that helps break up proteins; ammonia accumulates in blood
Jesse Gelsinger: volunteered for gene therapy at 18, died 4 days later |
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Cystic Fibrosis
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nonfunctional chloride ion transport protein (CFTR gene)
mucus build-up in lungs and other organs Inhalers: limited, short term sucess |
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requirements for approval of clinical gene therapy trials
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Knowledge of defect
Animal modal Sucess in human cells growing in vitro No alt. therapies Safe experiments |
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Mechanisms in gene therapy
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Ex vivo= genetic altering cells removed from patient and implanted back in
In situ= localized gene thearpy in easily accessible body part In vivo= direct genetic manipulation of cells in body |
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germline therapy
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alters DNA of gamete or fertilized ovum
heritable (not done w/ humans) |
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somatic gene therapy
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alters somatic cells, nonheritable
genes implants cells and tissues Correction may not be sufficient to overcome symptoms; immune system may attack altered cells |
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Sites of gene therapy
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Skin
Lungs Liver Cancer |
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delivery mechanisms
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Physical: electroporation, microinjection
Chemical: liposomes Biological: viruses |
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sickel cell anemia
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best understood genetic condition
Mutation, how phenotype arises, globin gene regulation |
