Biology 1002B Lecture Notes - Lecture 11: Embryonic Stem Cell, Petri Dish, Viral Vector

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Basic mechanisms of human gene therapies: viruses: using tissue specific viral vectors to deliver transgenes into the target tissue, example: using a viral vector to deliver normal cftr genes into the airway of patients with cf. Basic mechanism of crispr/cas9 mediated immunity in bacteria: Crispr: did not evolve as a genome editing system; began as curiosity research, mechanism that confers immunity from viral attack to single cell bacteria. Gene editing ; we can make essentially any change we want in any genomes. Fundamental ethic issues arising from human genome editing: unethical because we do not know how exactly changing the human genome will effect the life of the person. It is unethical to change heritable dna to make genetically modified children. Rationale, and basic process, for creating three parent babies : Rationale: when women carry mitochondrial disorders, all of their children will also have that disorder, mitochondrial diseases affect the bioenergetics of cells; cells cannot make enough energy.

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