Lecture 5 - Cf and small molecules II Flashcards
1
Q
heavy CFTR mr
A
mature and glycosylated
2
Q
Light CFTR mr
A
immature and not fully glycoylated
3
Q
F508del CFTR
A
remains immature and is sent for degradation due to misfolding
4
Q
Results of increasing lumacaftor concentration
A
increased number of mature CFTR in F508del mutations
5
Q
cAMP is used to
A
stimulate CFTR
6
Q
combination therapy is made up of three drugs…
A
Ivacafotr, Lumacaftor and Orkambi
7
Q
Combination therapy phase 3 trial results
A
increased FEV1%
all parameters better
Only a mild impact
reduced hospitalisations IV-AB treatment
8
Q
Combination therapy mechanism of action
A
can traffic F508del CFTR to the membrane so it is functional, however this is not sufficient to improve symptoms
But using potentiators to can enhance CFTR function and therefore relieve symptoms
