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UID:20250905T123341EDT-9259UZWcUI@132.216.98.100
DTSTAMP:20250905T163341Z
DESCRIPTION:Cystic Fibrosis is a recessive inherited disease\, that severel
 y affects more than 80.000 patients worldwide. Mutations in the CFTR gene\
 , encoding a transmembrane protein involved in transcellular chloride tran
 sport\, cause progressive pathology in pancreas\, liver\, gut and lung. De
 spite recent progress in development of CFTR targeted compounds\, an effec
 tive cure for all patients is not yet available.\n\nWhen the gene was clon
 ed in 1989\, it became clear that correction of CFTR deficiency was the pr
 ime objective. The most important target tissue being the airway epitheliu
 m and the required correction level some 10% of average normal. This was b
 ased on the consideration that known heterozygotes\, the parents of CF pat
 ients\, are healthy\, and that mutant variants with partial activity in fu
 nctional assays cause less or no pathology than inactive forms.\n\nInitial
 ly\, somatic gene therapy appeared the way forward. However\, this require
 d the development of novel technology that is still insufficiently effecti
 ve. Nevertheless\, new developments in stem cell technology and gene editi
 ng recently caused a revival of this field. As we learned more about the p
 roperties of the mutant CFTR forms that are found in a majority of CF pati
 ents (F508del and G551D)\, screening large compounds libraries for molecul
 es that correct their deficiency became the main approach.\n\nWe will disc
 uss the effect of CFTR deficiency\, not only on trans-epithelial ion and f
 luid transport\, but in particular on downstream processes that contribute
  to CF lung inflammation and tissue remodeling. Based on our recent studie
 s in CF infant lung and cell culture models\, we consider the contribution
  of oxidative stress and abnormal lipid metabolism\, and whether remission
  or prevention of pathology can be achieved with partial correction of mut
 ant CFTR activity in all target cells\, or by overexpressing normal CFTR i
 n part of the cells. Finally\, we will discuss opportunities for alternati
 ve or additional therapeutic targets.\n\nThis event is part of the Departm
 ent of Physiology Friday Seminar Series and is sponsored by the Cystic Fib
 rosis Translational Research Centre.\n
DTSTART:20191108T160000Z
DTEND:20191108T170000Z
LOCATION:Room 1034\, McIntyre Medical Building\, CA\, QC\, Montreal\, H3G 1
 Y6\, 3655 promenade Sir William Osler
SUMMARY:Seminar - Therapy of Cystic Fibrosis\, when\, what target\, and how
  much is enough?
URL:/physiology/channels/event/seminar-therapy-cystic-
 fibrosis-when-what-target-and-how-much-enough-300794
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