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14 Cards in this Set

  • Front
  • Back
Gene therapy
replaces malfunctioning gene, alleviates symptoms
breed better humans by encouraging reproduction of ppl with "good" genes and discouraging reproduction of ppl with "bad" genes
therapeutic vs. enhancement
therapeutic: attemt to cure a disease
enhancement: attempt to chose a child's tratis (stregnth, intelligence)
Immune system is nonfunctional (ADA deficiency, Xlinked)
only human disease to be cured by gene therapy
Xlinked SCIDS
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)
OTC trial
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
Cystic Fibrosis
nonfunctional chloride ion transport protein (CFTR gene)
mucus build-up in lungs and other organs
Inhalers: limited, short term sucess
requirements for approval of clinical gene therapy trials
Knowledge of defect
Animal modal
Sucess in human cells growing in vitro
No alt. therapies
Safe experiments
Mechanisms in gene therapy
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
germline therapy
alters DNA of gamete or fertilized ovum
heritable (not done w/ humans)
somatic gene therapy
alters somatic cells, nonheritable
genes implants cells and tissues

Correction may not be sufficient to overcome symptoms; immune system may attack altered cells
Sites of gene therapy
delivery mechanisms
Physical: electroporation, microinjection
Chemical: liposomes
Biological: viruses
sickel cell anemia
best understood genetic condition
Mutation, how phenotype arises, globin gene regulation