Term
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Definition
-invidual with two alles for a disorder will have phenotpic symptoms of disorder
-often produces protien that can't do its job
-can produce no protein at all |
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Term
| the problem with recessive disorders |
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Definition
-typically not making functional protein
-immune system may attack protein |
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Term
| solution with recessive disorders |
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Definition
-could replace specific part of protein that isn't working
-normally add a new piece of dna to chomosomes in cells--this DNA then will make a new protein
-fixed by gene replacement therapy |
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Term
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Definition
-need only one allele to have phenotpyic symptoms of disorder
-strong protein causes problems |
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Term
| problem with dominant disorders |
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Definition
| -often caused by a strong protein that causes problems whenever it is present |
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Term
| the solution to dominant disorders |
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Definition
-stop protein produciton
-fixed by RNA therapy |
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Term
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Definition
| liposomes and chemical linkage of DNA to special molecules is an example |
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Term
| gene therapy important steps and common approach |
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Definition
-an important step in sucessful gene therpy is delivery of DNA or RNA to target cells by a vector
-most common approach relies on inserting new gene in place of disease causing gene--new gene produces protein that replaces the function of the protein from the disease gene |
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Term
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Definition
-technique for correcting defective genes responsible for disease development
-normal gene may be inserted into nonspecific location withiin genome to replace faulty gene
-abnormal gene swapped for normal gene through homologous recombination
-abnormal gene repaired through selective reverse mutation
-regulation of particular gene could be altered |
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Term
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Definition
| -carry DNA or RNA to cell where it goes into chromoomes |
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Term
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Definition
-virus: protein shell carrying DNA or RNA
-liposomes: lipid sphere that carries DNA or RNA
-nanoparticle: very small particles that carry DNA or RNA
-nonviral options: direct introduction of DNA into target cells |
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Term
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Definition
-can cause immune response
-difficult to get enough genes to enough cells
-insertion of the genes on the chromosome can cause cancer |
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Term
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Definition
| immune systems enhanced response to invaders it has seen before makes it difficult for gene therapy to be repeated in patients |
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Term
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Definition
| -conditions or disorder that arise from mutations in a single gene are the best canidates for gene therapy |
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Term
| successful gene replacemtn therapy requires a gene with... |
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Definition
| a promoter that is expressed in the proper human tissue |
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Term
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Definition
| turns on transcrition--make piece of RNA and then transcribes into protein |
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Term
| steps in gene replacement therapy |
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Definition
-recombinant DNA is made by putting together a pormoter and a gene
-recombinant DNA is put into vector
-recombinant DNA is delivered to cells
-recombinant DNA becomes part of patients chromosomes
-recombinant DNA is trascribed and translated into a protein |
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Term
| which is least likely to cause an immune response to the gene therapy vector |
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Definition
| -removing cells from patient, using vector to deliver gene to cells in the lab, and replacing cells in the patient |
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