BIMM 110 Lecture Notes - Lecture 9: Complementary Dna, Base Pair, Jennifer Doudna
25 Feb 2017
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Vx-770 acts as a potentiator of cftr in recombinant cells. Identified through high throughput screening of 228,000 chemically diverse compounds: class iii (gating) mutation in 228k wells, each have a diff chemical membrane potential changes in well when function of. Cftr was restored color will change in the well. In january 2012, fda approved a new drug for patients with class iii (gating mutations) such as g551d (glycine aspartic acid) The drug called kalydeco (vx-770, ivacaftor) is a potentiator of cftr, improves cl- transport through the channel. Orkambi: combination of corrector (class ii) and potentiator (class iii, improvement of lung function only by 3% Delivering a healthy cftr gene into lung epithelial cells: viral methods of delivery, immune response; not enough cells transduced, not enough cftr produced. Adenoviruses: lung cells are their natural target. Adenoviral vectors (adv/aav): depleted of viral genes in their genomes, remain outside of the genome.
