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13 Cards in this Set

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Gene Therapy
-several steps
-production retroviral vector allowing entry therapeutic gene to patient`
Vector production
-in vitro, directly inoculated into patient
-genetically modified blood cells from patient in vitro, re-inoculated hopes will proliferate
Ex vivo gene therapy
-requires:
-vectors
-ancillary factors
-cells to target (autologous/allogeneic/xenogeneic)
Vectors
-need to deliver efficiently in cells
-retroviruses
-adenoviruses
-adeno-associated viruses
Ancillary factors
-cytokines
-growth factors
-hormones
Target cells
-fibroblasts
-heptocysts (for hepatocellular carcinoma)
-hematopoeietic cells (easily removed and genetically modified)
-keratinocytes
-myoblasts
-stem cells
Retro-vectors
-needs essential info for functional virus: gal, pol, env, LTRs
-psi sequence: packaging RNA into genome
-minimal: LTR, PBS, Psi, extended packaging sequence to ensure RNA incorporated to retroviral vector
proviral DNA gene therapy into tissues
-minimal: LTR-psi-cDNA-LTR
-vector because cell its going into has gag, pol and env, its a packaging cell line
Packaging Cell Line
-independently engineered so expresses gag, pol and env
-completely different genes cassettes or an integrated proviral DNA
-transfect DNA into cell, gag/pol/env in cell help with repliaction cDNA construct and help to produce RNA
-used to make a population of a virus to be purified and get retrovirus stock
Neo
-selectable marker
-drug resistance, fluorescence
-allows for selection of cells successfully expressing transgene
-in vitro
-used in sequences that express two genes
cDNA
-therapeutic gene in sequences that express two genes
Gene delivery
-using retroviral vector
-CMV-RU5'-Psi-transgene-GFP-U3R
-transfect into packaging cell line
-integration occurs giving retroviral constrcut, transcribed to give mRNA spliced
Retrovirus stock
-of replication defective retroviruses