Personalized medicine and gene therapy

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13 Terms

1
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Pharmacogenomics

identifies responders and non responders to different treatments, casual genes and biomarkers to see who responds to treatment

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Pharmacokinetics

deals with how the body processes the drug (ADME) (absorption distribution metabolism excretion)

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Pharmacodynamics

Activity of the drug in the body

4
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Warfarin

anticoagulant that inhibits vit k synthesis factors

(larger window for therapeutic effect)

variation in individual therapy requiring for a response

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SNPS in metabolic and target proteins determining effective warfarin dose

CYP450 enzyme that found in many drugs, SNPS with this enzyme can alter metabolism and decrease dose

VKORC1 is the target that has many SNPS that be sensitive

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Pharmacogenetic testing

Blood tests can be ordered via doctors to test metabolization

Biopsies of cancer tissue needed

DTC available for drugs

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Molecular etiology of Cystic fibrosis

CTFR ABC transposon > anion channel conducts chloride and bicarbonate ions out of cell > mutations cause CFTR linked to development of CF

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Cf therapeutics (f508 deletions)

functional channel misfolding resulting in degradation

treated with fold corrector

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cf therapuetic (early stop codon)

needs to promote stop codon readthrough

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Pharmacogenetic testing

blood tests can be ordered to see how metabolization works

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Genotyping and breast cancer

The types of gene received can determine whether hormonal therapy would work

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RNA based gene therapy

addresses diseases caused by mutation o target degradation of the non-functional copy of the gene. viral diseases by targeting viral RNA

cancer treatment, can target undruggable genes, doesn’t change the host genome

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Antisense oligonucleotides therapy

results in less protein production using RNA interference (cleaving mRNA)