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7 Cards in this Set
- Front
- Back
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3 requirements for gene tx of monogenic dz
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1. mutation in single gene responsible for dz
2. gene constitutively expressed 3. overexpression is nontoxic |
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List 5 advantages of retroviral/lentiviral vectors for gene therapy
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Efficient transduction of lymphocytes
Lentivirus can transduce non-dividing cells Large transgene packaging capacity Stable integration of provirus into host genome Essentially non- immunogenic |
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List 4 disadvantages of retroviral/lentiviral vectors
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Insertional mutagenesis (leukemia)
Retrovirus can’t transduce nondividing cells Relatively low titers Potential for generation of replication competent virus |
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4 advantages of adenovirus vectors
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Large transgene packaging capacity
Highest titers of all viral vectors Able to infect a wide variety of tissues transgenes expressed to high levels |
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5 disadvantages of adenovirus vectors
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Preexisting immunity
Vector dose dependent inflammatory response Relatively short duration of gene expression Genome is not stably integrated into patient genome Potential for generation of replication competent virus |
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5 advantages of adeno-assoc'd vectors
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Not associated with any known disease
Site specific integration in Chrom19 Broad tissue tropism Transduction of nondividing cells Prolonged gene expression |
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4 disadvantages of adeno-assoc'd vectors
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Small transgene packaging capacity
Gene expression is not observed for weeks after transduction Moderate to low titers Immune responses to the capsid can limit readministration |
