Thursday 21 March 17:10-18:40
Special Group Discussion 1 - CFTR Gene Expression / Modifiers
Moderators: Harriet Corvol (FR) / Paul McCray (US)
A focus of this session will be to have an interactive discussion regarding the limitations in knowledge in our understanding related to the regulation of CFTR expression and modifiers of CFTR expression and function. From this discussion we will generate suggestions from future research directions and priorities. The goals include improved understanding of factors influencing CFTR expression and function, and how genetic modifiers influence phenotype. Concepts that may lead to new therapies will be discussed. Our emphasis will be driven by audience participation and input.
This workshop will look at new developments in the expression and purification of CFTR. This will include discussion of the domains of CFTR (nucleotide binding domains 1 and 2, R region) as well as for the full-length protein which has to be solubilised from the membrane and purified in the presence of a detergent. The workshop will take the form of short 'current state of the art' methodology presentations, describing a range of the available expression systems and the particular nuances of optimising the expression of CFTR domains or full length proteins. The workshop will then discuss the latest methods for purification of CFTR domains and full length protein and the barriers than remain to be overcome. Both parts of the workshop will be followed by question and answer and troubleshooting sessions. The presenters will be encouraged to use movies or videos to illustrate the methodology as well as handout sheets and slides. Postdoctoral researchers and PhD students from various labs will be encouraged to present and act as discussants in the workshop.
Saturday 23 March 11:00-12:30
This discussion will cover three topics, with short introduction talks that are intended to promote active general discussion by the audience. The first topic will focus on the search for new correctors using new methodolgies stemming from recent progress in our understanding CFTR folding and its dependence on both NBD1 stability and domain interactions. This will be followed by a discussion of potientiators and the mechanism of action VX770, focusing on the role of nucleotides and phosphorylation. We will conclude with discussion of novel in-vitro assays of CFTR modulators.
Workshop II - Imaging - Microscopy
Moderators: Peter Haggie (US) / Rob Tarran (US)