BIMS 201 Lecture 10: treatment of genetic diseases and lysosomal storage disease
11 May 2017
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Document Summary
Treatment of genetic diseases: o ver time, o ver the years, treatment of genetic diseases have evolved with new technologies available. In order of sophistication: r emoving an affected body part, affects phenotype, r eplacing an affected body part or biochemical with material from a donor, e . x. Hg h: human growth hormone therapy: affects phenotype. Yeast have the benefit of being able to properly process protein since it is an eukaryote. C on: cost: g ene therapy, r eplace mutant alleles, attempts to affect genotype. S omatic: attempts to treat the disease t the source, the bad alleles in the cells that are affected, g ermline. C orrect the disease in future generations as well: not approved as of now, human g rowth hormone (hg h) is deficient in pituitary dwarfism. Individuals get the hormone from cadavers (in past) P urifying a hormone from cadavers -> intro of diseases like.
