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10 Cards in this Set
- Front
- Back
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what is hematopoietic transplantation?
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bone marrow transplant
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What is Seed threshold?
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you have to get enough of the stem cells in the body for them to be stable and proliferate
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how is cell culture expansion a challenge to stem cell use?
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when you are replicated stem cells to get enough to use, they start to differentiate, making them no longer useful
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What is adult stem cell therapy?
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take undifferentiated (these are still more differentiated than embryonic stem cells)
MESENCHYMAL CELLS out of your body |
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What are the 2 forms of gene therapy?
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Somatic transformation: deliver a wild type gene to somatic cells of an adult or child, without eliminating the mutant allele
Germ Line Transformation: replace a mutant gene with a wild type allele in the germ line (microinjection)--USED FOR RESEARCH ONLY |
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what is ex vivo gene therapy
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take cells out
modify them inject them back in |
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indirect gene therapies are used for what? examples?
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to treat cancer
immune response: antigenic genes, transformed cells cleared Suicide genes: gene expression kills transformed cells Tumor suppressors: genes that inhibit tumorgenesis (p53) |
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Challenges of gene therapy?
(objective-just read) |
Transformation level
Expression level Regulation Targeting to specific organs Transformation Stability |
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Major danger in gene therapy?
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Immunologic reaction--can lead to death
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in ADA deficient SCIDs, what was the key to making gene therapy work?
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they did ex vivo implantation
but the KEY IS TO KILL PRE-EXISTING T LYMPHOCYTES so that the new ones have room to grow in X linked form, the pts developed activation of oncogenes (womp womp) |
