RT Book, Section
A1 Sorscher, Eric J.
A2 Jameson, J. Larry
A2 Fauci, Anthony S.
A2 Kasper, Dennis L.
A2 Hauser, Stephen L.
A2 Longo, Dan L.
A2 Loscalzo, Joseph
SR Print(0)
ID 1162605891
T1 Cystic Fibrosis
T2 Harrison's Principles of Internal Medicine, 20e
YR 2018
FD 2018
PB McGraw-Hill Education
PP New York, NY
SN 9781259644016
LK accesspharmacy.mhmedical.com/content.aspx?aid=1162605891
RD 2023/02/08
AB Cystic  fibrosis  (CF)  is  an  autosomal  recessive  exocrinopathy  affecting  multiple  epithelial  tissues.  The  gene  product  responsible  for  CF  (the  cystic  fibrosis  transmembrane  conductance  regulator  [CFTR])  serves  as  an  anion  channel  in  the  apical  (luminal)  plasma  membranes  of  epithelial  cells  and  regulates  volume  and  composition  of  exocrine  secretion.  An  increasingly  sophisticated  understanding  of  CFTR  molecular  genetics  and  membrane  protein  biochemistry  has  facilitated  CF  drug  discovery,  with  a  number  of  new  agents  recently  approved  or  advancing  through  the  clinical  testing  phase.