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| Frage | Antworten |
| current molecular therapies undergoing trials for cancer treatment | stem cell therapy. cytotoxic drugs. manipulating transcription. using abs bound to toxins. personal vaccines. recombinant proteins. turning off oncogenes. tumour suppressor genes |
| Sleeping beauty transposons; a non-viral vector for gene therapy | provides only transient expression of a transgene from a promoter unless transposed into a host genome. Delivery byelectroporation or transfection or. hydrodynamic injection |
| major problem with adenoviruses as vectors | multiple doses needed coat protiens highly immunogenic |
| advantages of retroviruses as viral vectors | up to 7 kb of DNA can be carried once integrated, will replicate with cell cure if stem cells targeted less immunogenic |
| disadvantages of retroviruses as vectors | random integration may disrupt genes difficult to grow only infects dividing cells |
| advantages and disadvantages of adenoassociated viruses as vectors | effeciant delivery of DNA specific to chromosome 19 but needs other viruses to complete life cycle |
| somatic germline therapy | treats somatic cells change cannot be inherited |
| germline therapy | illegal therapy on germ cells therfore changes can be passed on |
| 2 types of gene therapy | gene augmentation gene silencing |
| liposomes as vector | reletively effecient targeting a problem could have reaction to liposomes |
| antibodies as vectors | can be used with liposomes may help overcome targeting problems |
| particle bombardment as vector | prob only good for skin transient expression low effeciency |
| ligands as vectors | targeted delivery transient expression contains antibodies and growth factors |
| take home message re gene augmentation therapy | not really successful due to lack of vector systems once this is overcome and DNA expressed, things like cytsic fibrosis curable |
| tissues targeted for gene augmentation therapy | lungs bone marrow liver brain muscle |
| major problems with gene augmentation therapy | only used for recessive disorders damage to organs cannot be undone so targeted at the very young only helps affected individual dangerous/experimental |
| criteria for gene therapy | life threatening condition no other tx available gene cloned target organ accessible minimal expression gives relief |
| what type of viral vectors available for gene therapy? | adenoviruses adeno associated viruses retroviruses |
| non viral vectors for gene therapy | naked DNA plasmids particle bombardment liposomes ligands |
| problems with human organs for donations | supply HLA matching immunosupression |
| problems with animal organs for human use | anatomy physiology immunosupression |
| what are the general problems with organs for donation | immunogenecity HLA rejections xenozoonoses |
| what types of recombinant protiens may be used in theraputic strategies? | 1. enzymes 2. clotting factors 3. hormones 4. cytokines 5. growth factors 6. antibodies |
| adenoviruses as vectors | natural infectios agents infect dividing and non dividing cells can carry 7.5kb if E1+E3 genes removed do not integrate into host genome therofre expression transient no E1= safe |
| how to engineer transgenic pig for xenotransplantation | 1. change Gal-alpha1-3Gal by knockout or by adding alpha-2-fucosyltransferase 2. add human CD59 +human membrance cofactor +human DAF |
| 5 molecular therapies for the treatment of cancer | 1. recombinant protein therapy 2. organ transplant 3. stem cells 4. immunotherapy 5. gene therapy |
| an example of recombinant protien therapy for cancer treatment | cytokines for lymphoma patients |
| an example of organ transplant for cancer treatment | new liver |
| an example of how stem cells are used for cancer treatment | use of blood derived haemopoeitic stem cells for transplant in leukaemia |
| describe reproductive stem cell therapy | 1. an enucleated egg and genetic material from an adult cell are combined 2. new egg construct implanted 3. allowed to come to full term illegal in UK |
| describe theraputic stem cell therapy | 1. ennucleate egg and genetic material from adult cell combined 2. new egg construct grown to blastula stage then disrupted 3. stem cell yield grown in lab legal in UK under license |
| list 6 potential problems associated with production of a theraputic monoclonal antibody | 1. choice of organism ie prokaryote v eukaryote 2. glycoslyation 3. scale up 4. costs 5. authenticity 6. tests for contamination |
| name 6 vector properties important in human gene therapy | 1. easy transfection into host 2. gene stability 3. adequate expression of gene= good production in host 4. easy to make/store/validate 5. no disease/immune response in patient 6. non toxic |
| what is a SNP? (single nucleotide polymorphism) | small genetic change/variation occurs in 1% of population a single nucleotide replaces one of the other 3 involves only 2 alleles varies with racial background |
| Where do most SNPS occur? | outside of coding sequence |
| what happens when a SNP occurs within the coding sequence? | alters the function of a protein position is important as effects expression |
| how often to SNPs occur? | on average every 500-200 bp |
| how many common SNPs are there? | estimated 240,000-400,000 |
| what responses do SNPs affect? (5) | drugs pathogens vaccines chemicals food |
| how are SNPs useful in drug therapy? | understanding differences in drug absorption + clearance why some ppl have side effects could lead to designer drugs |
| how would poor and rapid metabolisers respond to a prodrug like codiene? | poor: poor response/drug accumulation rapid: good response/rapid effect |
| how would poor/rapid metabolisers respond to an active drug like omeprazole? | poor: good response/ ? need a lower dose rapid: poor response / may need incresed or slow release dose |
| what would be the impact of global testing for SNPs? | too costly but would save money in long term personalised medicine improves results less drugs sold so big pharma would not support |
| antidepressants and SNPs | many people affected w/depression many antidepressants dont work but ppl afraid to say that=thought to be mad huge problem preventable side effects can be worse than condition |
| problems associate with production of recombinant protiens (9) | 1. host authenticity 2. vector authenticity/stability 3. insert stability 4. fermentation techniques 5. product production 6. post-translational modifications 7. activity 8. toxicity 9. QA/QC |
| what are 2 vector types for human gene therapy? | viral non viral |
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