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GEN #18 Gene Therapy I unfinished
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The manipulation of an individuals genomic information to produce a therapeutic outcome is _______
gene therapy
A liver disease that cause high ammonia levels and is abbreviated OTC is
ornithine transcarbamylase deficiency
ADA deficiency causes
severe combined immunodeficiency syndrome
Ways to treat acquired diseases are
Gene augmentation therapy
Eliminate selected cell populations
Correct an acquired gene mutation
Gene augmentation therapy refers to an acquired ______ function
loss of gene
Generally the introduction of_______will overcome a loss of gene defect
a single gene
___________ removes a specific cell population from the body
Elimination of Selected Cells
Inserting a gene to code for a cellular toxin is an example of ________
Elimination of Selected Cells
Direct killing of diseased cells can be done by inserting a __________ or a ___________
toxin gene
prodrug gene
Prodrug activation therapy requires viral _________ to be transfected into cells
thymidine kinase
ganciclovir is ___________ by the viral TK
monophosphorylated
_________ finish adding phosphate and ganciclovir blocks cell division.
host cells
ganciclovir monophosphate enters adjacent cells through ______ formed by ________
gap junctions
connexon proteins
ganciclovir monophosphate is ________ in the adjacent cell and kills that cell as well
triphosphorylated
_________ substitutes a wild-type gene in place of a gene that has been altered due to chemical, viral, or physical injury
Correction of a Gene Mutation
Correction of a Gene Mutation uses __________ to do replacement
homologous recombination
Targeted Gene Editing disrupts a single gene using a _______ approach
zinc finger nuclease
zinc finger nuclease is an engineered fusion protein containing
DNA cleavage domain of the restriction endonuclease Fokl
Zf DNA binding domain
Fokl needs to ______for activity using two ________ proteins
dimerize
nonpalindromic
If DNA binding domain contains ________ covering ___ zinc finger domains, will provide unique specificity of binding.
18 bp
3
2 most popular viral systems are
Retroviral vectors
Adenoviral vectors
Author
HUSOP2014
ID
143928
Card Set
GEN #18 Gene Therapy I unfinished
Description
GEN #18 Gene Therapy I
Updated
2012-03-26T15:38:50Z
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