Term
|
Definition
| unmethylated CpG DNA and dsRNA and degraded products |
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Term
| unmethylated CpG DNA and dsRNA immuno genicity |
|
Definition
| issue with nonviral gene therapy |
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Term
|
Definition
| using siRNA to knockdown gene that is known to increase circulating cholesterol |
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|
Term
| RNAi approach to target PCSK9 |
|
Definition
| example of nonviral gene therapy |
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|
Term
| protecting siRNA from nuclease degredation |
|
Definition
| use of lipidoid formulation allowing expression of siRNA in liver with minimal toxicity |
|
|
Term
| transient effect of RNAi nonviral approach targeting PCSK9 |
|
Definition
| overtime the lowering effects of LDLs diminished |
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Term
|
Definition
| evolved to deliver DNA over millions of years, can be modified to carry genetic payload, and we can remove viral DNA |
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Term
|
Definition
| only effective method for HSC gene therapy |
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|
Term
| integration of viral DNA that is carried over to daughter cells |
|
Definition
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|
Term
| foamy retrovirus structure |
|
Definition
| LTR, Gag, Pol, Env, tas, LTR |
|
|
Term
| gamma retrovirus structure |
|
Definition
|
|
Term
| Lentivirus (HIV-1) retrovirus structure |
|
Definition
| LTR, Gag, Pol, vif, vpu, Env, LTR |
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Term
|
Definition
| gene involved in Adrenoleukodystrophy that codes for protein that transports very long chain fatty acids to peroxisome where fatty acid degradation occurs |
|
|
Term
| gene therapy for adrenoleukodystrophy |
|
Definition
| using autologous HSC transplant in patients that have no HLA matches (for allogenic HSC transplant), lentiviral vectors are used to correct ABCD1 mutation |
|
|
Term
| foamy virus integration within host DNA |
|
Definition
| less frequently near proto-oncogenes than MLV (gamma) or HIV vectors |
|
|
Term
| foamy transduction of Hematopoietic progenitors and mesenchymal stem cells (MSCs) |
|
Definition
|
|
Term
| Anti-AIDS stem cell gene therapy |
|
Definition
| transduction of anti-HIV genes using fomay retrovirus vector |
|
|
Term
| viral gene therapy targeting postmitotic tissue |
|
Definition
| lentiviruses and adeno-associated vectors |
|
|
Term
| foamy (gamma) viruses for postmitotic tissue gene therapy |
|
Definition
| are inefficient because they require mitosis that does not occur as frequently in these types of tissues |
|
|
Term
| adenoviruses for postmitotic tissue gene therapy |
|
Definition
| causes inflammatory response |
|
|
Term
| using blood cells as gene therapy target |
|
Definition
| can use ex vivo gene delivery, low vector particle numbers needed, integration vectors MUST be used due to expansion characteristics of HSC |
|
|
Term
| use of muscle cells as gene therapy targets |
|
Definition
| must be transduced in vivo, needs high vector particle numbers, episomal vectors can be used due to the lack of cell division |
|
|
Term
| adeno-associated viruses (AAV) for targeting muscle cells |
|
Definition
| efficient target cell, persist long-term episomes, do not have site-specific integration, they do integrate at dsDNA breaks, long term transgene expression, illicit minimal immune response |
|
|
Term
| AAV vector expressing RPE65 |
|
Definition
| injected into retina of dogs/humans that have leber's congenital amaurosis 2 and showed no adverse events |
|
|
Term
| advantages of Lentiviral vectors |
|
Definition
| replication-integration, integrates efficiently, transmission of selected gene into daughter cells, good for cells that have expansion capabilities, moderate titers of viral particles needed |
|
|
Term
|
Definition
| AAV gene therapy not effective for dogs, larger mammal model |
|
|
Term
| advantages of AAV vectors |
|
Definition
| replication-incompetent, inefficient integration, persists as episome, good for tissues that do not replicate, very high titer of viral particles needed |
|
|
Term
| chimeric antigen receptor genes induced to T cells |
|
Definition
| artificial T cell receptors that redirect the T cells to kill leukemic CD 19 expressing B cells |
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|
Term
| oncolytic pox virus Jx-594 |
|
Definition
| virus designed to specifically replicate in cancer cells (that express TK in high numbers) and kill them and stimulate anti-tumor immunity |
|
|
Term
| poxvirus (vaccinia) natural abilities |
|
Definition
| resist serum inactivation after intravenous administration, spread to distant tissue |
|
|
Term
| major different with the use of Poxvirus as vector |
|
Definition
| infects AND replicates within tumor cells |
|
|
Term
| SCID-X1 gene therapy trial |
|
Definition
| lead to dysregulation of a proto-oncogene LMO2 and vector-mediated leukemia in some patients, typically through enhancer-mediated activation |
|
|
Term
| major issue with integrating viruses |
|
Definition
| may alter host gene expression |
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