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Platforms

The program will develop resources accessible by the CF research community to advance the development of precision medicine to treat CF:

1. Genetics Resource

Genome sequencing and gene expression profiles from the set of 100 CF patients were collected in conjunction with certain clinical data that can be used to study how the presence of modifier genes influences the severity of CF disease and will lead to the development of new therapies and treatments.

2. Stem cell Resource

A bank of iPS (induced pluripotent stem) cell lines from the same 100 unique CF patients that were selected are accessible to CF researchers across Canada.  These iPS cells are a renewable source of patient-specific cells that can be differentiated into virtually any cell type that is affected by CF, such as ciliated lung epithelium, bile duct cells and pancreatic progenitors. Thus iPS cells provide a unique opportunity to explore the correction of CFTR defects across a wide range of mutations on different genetic backgrounds and in different disease-relevant cell types.

We will also be creating a bank of primary nasal tissue from the same 100 CF patients that can be cultured much like lung cells.  The nasal tissue will be studied to show the CFTR function and characteristics of this native cell type from the same patient.  We plan to make the nasal cell bank available to the wider CF research community in the future.   

3. Drug Profiling Resource

We plan to offer in the future in vitro pre-clinical trials for defining the number of potentially responsive patients to a new compound by taking advantage of functional assays employing the 100 patient bank of the stem cell resource.  This will help enable researchers to develop precision medicine for CF.  

4. Clinical Phenotyping Resource

We will use our extensive experience in CF clinical trials and the resources above to provide leadership and training in strategic clinical trial design with associated in vitro functional assays to help advance potential new therapies to treat CF to the clinic and identify how these treatments can lead to precision medicine to best treat each person’s CF.     

For more information on obtaining access to the above resources, please contact Paul Eckford, Program Manager, at peckford@sickkids.ca.