INTER-LS 101 Lecture Notes - Lecture 34: Eye Disease, Drug Delivery, Rhodopsin
7 Dec 2016
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Biology of vision - lecture 34 - treatment of eye diseases (11/30/16) Many inherited eye diseases cause retinal degeneration. Goal is to rescue cells that are still alive. - study test on animals to check safety and side effects: fully evaluate effectiveness of treatment, larger # of patients, eye disease has small patient population - setback. 1990 - 1st gene mutation found in rhodopsin gene. Defective gene can be replaced with pure gene. Gene therapy: introduce genetic material into organism to slow, prevent, or reverse progression of a disease. Preventing destruction of introduced gene by immune system. Targets actual cause of disease, not symptoms. Expression of desired gene may exceed duration of action of drugs. Expression can be regulated by turning gene on or off. Slow or prevent degeneration -- doesn"t cure. Some forms of rp and slowed with vitamin a.
