BIOL SCI 150 Study Guide - Midterm Guide: Adenosine Deaminase, Multiple Organ Dysfunction Syndrome, Rpe65

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Name a few methods of gene therapy: viral delivery of a gene, viral delivery of rnai to shut off a gene, viral delivery of gene editing enzymes to inactivate a gene. Differences between three types of virus vectors: adenovirus, modified dna is injected into adenovirus vector which binds to cell membrane and is transported across in a vesicle. Injects the new gene into nucleus and cell makes protein. Is not integrated into genome - so no risk of mutation. Cell will produce protein but descendants of the cell will not produce protein so it needs multiple treatments since cells are constantly renewing. b: risk of unwanted out of control immune responses i. ii. In 1999, a patient was being treated w/ an adenovirus and went into multi-organ failure. So when retrovirus infects host cell, it inserts rna material and some reverse transcriptase enzymes, so the host cell can turn the rna into dna.