WVSOM -- Current and Future Medical uses of Molecular Biology
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| How is durg selection and dosing optimized? | using genetic information from genotype and phenoype. Mostly used in psychology.
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| What will a clinician found out by doing a genetic genotype/phenotype test? | To find out what kind of metabolizer they are.
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| What kind of conditions can be used to find genetic suceptiblity with an amplitude test? | Many types of cancer
Type 2 Diabetes
Alzheimer's disease
Osteoporosis
Infertility
Cardiovascular risk
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| You patient is found to have a highly methylated p53 gene. Do these results suggest that the patient has an increased or decreased risk of developed cancer? | Increased chance
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| What is epigenetics? | heritable changes in gene expression caused by mechanisms other than changes in teh DNA sequence
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| What are examples of epigenetics? | Histone modifications ("histone code")
DNA methylation
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| What is epigenetic testing mostly used for? | Prostrate cancer
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| What is gene therapy? | Correct genetic disorders by introducing a normal functioning copy of the defective gene.
No approved/marketed gene tehreapies or RNAi therapies
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| What has to happen for gene therapy to work? | the gene needs to be introduced into and expresssed in the correct cells at teh correct time
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| How do you introduce a gene into a human somatic cells? | In vivo and Ex Vivo
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| What is in vivo introduction? | Delivery method must be targeted to specific tissue.
Methods include injection or gene gun, viral vectors.
"Naked" DNA is very quickly chopped up in teh body so vehicles are used (lipsomes, nanoparticles, etc.)
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| What is Ex vivo? | Cells are removed form a patient and the gene is introduced.
Cells are then transplanted back into the patient.
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| Why use viral vectors? | We can manipulate viruses in the lab and introduce human genes. Viruses then inject their genetic material into the host cell as part of their life cycle.
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| What are the most common used viruses used? | Retroviruses, adenoviruses, adeno-associated viruses and herpes simplex virus 1
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| What must be done to viruses to be used in gene therapy? | The virus must be engineered to be replication defective. It can infect the cell but not replicate itself
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| What is retroviral vectors | The gene is integrated into teh host genome so daughter cells will have it.
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| What is the risk of retroviral vectors? | insertional mutagenesis
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| Can transponons be used? | yes, they are string to use them
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| What is episomal replication? | viral DNA does not integrate into the hose genome.
DNA is not passed on.
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| What are the problems associated with gene therapy? (6) | Multiple rounds of therapy often needed because it does not stay around long enough to be effective.
Immune responses can occur.
Potential for reactivation of virus.
Its hard to control DNA!!!! what if it integrates into a critical gene.
Disorders cau
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| What is epigenetics? | heritable changes in gene expression caused by mechanisms other than changes in DNA sequences
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| What is the current epigenetic test being used for? | prostrate cancer
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| What are the problems with gene therapy? | Immune responses can occur.
Multiple rounds may have to be used because DNA does not stay around long enough.
Its hard to control DNA
Potential for reactivation of viruses.
Expensive
Disorders with multiple genetic changes are more difficult to treat
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| What is stem cells? | undifferentiated cells with the capacity for self-renewal and the ability to develop into differentiated cells
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| Characteristics of embryonic stem cells | extemely versitile
infiite life span
ethical issues
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| totipotent | cells produced by the first gfew divisions of a fertilized egg
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| Pluripotent | isolated from teh inner cell mass of a blastocyst
can become any cell type except extraembryonic cells
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| What are sources of pluripotent stem cells? | available stem cell "line"
aborted, miscarried or donated IVF embryos
somatic cell nuclear transfer
altered nuclear transfer
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| What is SCNT | Somatic cell nucleear transfer (cloning)
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| How do you get stem cells to become what you want them to become? | exposure to the correct environment and growth factors.
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| What is a commonly used for of stem cell therapy? | bone marrow transplantation
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| What is autologous bone marrow transplant? | patients transplanted with their own stem cells
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| what is allogenic bone marrow transplantation? | patients transplanted with stem cells froma matched donor
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| You decide to bank the umbilical cord blood of your newborn child. The stem cells in the cord blood are most likely to be? | Pluripotent
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| What are the problems associated with stem ce3lls and gene therapy as therapeutics? | manufacturing and product delivery
incredibly expensive
technically difficult so only at a few sites.
Increased risk of cancer
Other unkown risks
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