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Cystic Fibrosis Centre

Future directions

  • Understanding the primary defect and identifying therapeutic targets through genetics, cell biology, structural and chemical biology.
  • Therapy discovery - identification of small molecule modulators of mutant CFTR by screening chemical libraries, development of peptide-based therapies.
  • Implementing pre-clinical trials on mouse models of CF and pre-clinical trials on primary human respiratory cells.
  • Developing meaningful clinical outcome assays in CF patients.
  • Bringing Discoveries to the Clinic: multi-centre efficacy trials with SickKids leadership both in Canada and in collaboration with the U.S. network (Therapeutics Development Network) as well as with Australian and European networks.
  • Conducting genetic counselling and training for parents and adults with both classic and mild CF.
  • Design and test treatments that prevent damage to the vulnerable infant. Developing assays of CF disease in newborns, infants and young children.
  • Combining knowledge of specific CFTR mutations and modifier gene variants with novel therapeutic discoveries to lead the way towards personalized medical treatment based on an individual patient's genotype and phenotypic response.