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| Question | Answer |
| how do we manipulate siRNA to make useful in theraputics | engineered into short hairpins structures which are more stable and provide longer gene supression via mRNA degradation |
| why are scientists interested in recombinant and DNA vaccines | 1. potentially safer 2. stability 3. reduced scale up time |
| why should we consider transplantation as gene therapy | you are replacing bad genes with good ones including all control mechanisms intact |
| what are the main issues when using stem cells for theraputics | 1. isolation from adult tissue 2. growth/differentiation in culture 3. cell stability 4. cell normality 5. safety 6. rejection 7. will cells age quicker 7. correct theraputic dosing |
| what types of vectors are used in gene augmentation therapy?- usually use | 1. viruses 2. liposomes 3. growth factors/antibodies |
| what types of vectors are used in gene augmentation therapy?- can also use use | 1. calcium phosphate precipitation 2. electroporation 3. microinjection 4.particle bombardment |
| outline the types of molecules used in gene silencing | 1. antisense 2. triple helices 3. ribozymes |
| anti sense molecules | short ssDNA pieces that bind to mRNA and prevent translation |
| triple helices | a molecule that could bind to DNA in the chromosome and turn the gene off |
| ribozymes | naturally occuring RNA sequences that cleave RNA to which they hybridise |
| what are the properties of vectors that are important in gene therapy | 1. transfection ability 2. gene carrying capacity 3. potential immune response 4. cell tropism 5. integration potential 6. disruption potential 7. targeting effeciency |
| what is a cybrid? | 1. An artificial hybrid cell produced by introducing nuclear material from one organism into a cell (of the same or different species) from which the nucleus has been removed |
| cybrid | is a eukaryotic cell produced by the fusion of a whole cell with a cytoplast. |
| what are the problems associated with the use of theraputic recombinant proteins | 1.cost 2. infections 3. activity 4. immune response 5. bioavailability 6. toxicity 7. stability |
| outline the genetic modifications of transgenic pigs engineered for xenotransplantation | 1. change Gal alpha 1-3 Gal by knockout or addition of alpha-2-fucosyltransferase 2. add human: CD59, membrane cofactor protein, DAF |
| list with examples the types of molecular therapies available for the treatment of cancer (5) | 1. recombinant protein therapy (cytokines) 2. transplant (liver) 3. stem cells (bone marrow transplant) 4. immunotherapy (HER2) 5. gene therapy (gancyclovir) |
| differentiate between reproductive and theraputic stem cell therapy | 1. both require enucleate egg plus DNA from adult cell 2. reproductive= new egg construct implanted and allowed to come to term- illegal in UK 3. theraputic= new egg construct inturruped at blastula to yeild stem cells to be grown in culture- legal in UK with license |
| list the problems associated with the use of monoclonal antibodies (5) | 1. acute reactions 2. antigenic modulation of tumour 3. tissue cross-reactivity 4. limited effector function 5. tumour localisation and penetration |
| list problems associated w/production of mAB | 1. choice of organism 2. glycosylation 3. scale up 4. cost 5. authenticity 6. test for contamination |
| what are the properties of vectors that are important in human gene therapy (8) | 1. easy transfection 2. gene stability 3. adequate expression of gene 4. adequate production of protein 5. easy to make/store/validate 6. not cause disease in host 7. not cause immune response 8. not be toxic |
| list the types of recombinant proteins that may be used in theraputic strategies + examples (6) | 1. enzymes 2. clotting factors 3. homones 4. cytokines 5. growth factors 6. antibodies |
| C/C two methods of producing transgenic animals- ES cells | 1.one generation 2. injection of stem cells 3. gene uptake checked in advance METHOD 1. isolate stem cells from embryo 2. insert desired gene into cells 3. cells inserted into host embryo 4. test for transgenes 5. activate embryonic development |
| C/C transgenic animal production-direct injection | 1. two generations 2. injection into egg 3. cannot be checked in advance METHOD 1. transgene expression cassettes inserted into plasmids, cloned and thranfected into cells 2. transgenes introduced to pronucei 3. embryos implanted 4. offspring tested 5. chimeras mated |
| list with examples the types of molecular therapies available for the treatment of inherited diseases (8) | 1. recombinant proteins 2. vaccines 3. stem cells 4. gene augmentation 5. antisense 6. RNAi 7. transplants 8. xenotransplants |
| briefly describe the types of vaccines used in humans | 1. whole organisms 2. sugars and proteins of organisms 3. recombinant proteins 4. DNA vaccines |
| list the problems associated with production of a recombinant theraputic protein (9) | 1. host authenticity 2. vector authenticity/stability 3. insert stability 4. fermentation techniques 5. product production 6. post-translational mods 7. activity 8. toxicity 9. QA/QC |
| vectors for human gene therapy-viruses | 1. adeno 2. retro 3 adeno-associated |
| vectors for human gene therapy- non viral | 1. naked DNA 2. plasmids 3. particle bombardment 4.liposomes 5. ligands |
| list the problems associated with using human organs for transplant | 1. supply/demand 2. HLA 3. immunosupression 4.infection 5. cancer |
| list the problems associated with using animal organs for transplant | 1. anatomy 2. physiology 3. immunosuppression 4. infection 5. unknown hormone interactions |
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