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Therapeutic applications

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High-throughput platform:
Dr. Daniela Rotin and colleagues created a high-throughput platform which enabled the identification of novel cellular proteins which act to correct the basic defect in the mutant: ∆F508-CFTR. Several of these cellular proteins are already known to be targeted by FDA-approved drugs (six in total) and these drugs are suitable for future validation in preclinical and clinical assays.

Status: Corporate Ventures (SickKids) is pursuing commercialization of cell lines used in robotic assays
(Daniela Rotin and SIDNET)

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Therapeutic short peptides:
Drs. Christine Bear and Charles Deber have designed short peptides which can reach the intracellular mutant protein and promote its trafficking and function at the cell surface. The potential of these peptides to act as therapies will be enhanced in our future work.

Status: Corporate Ventures (SickKids) is pursuing opportunities for development of therapeutic peptides
(P. Kim Chiaw, J. Cheung, C.M. Deber and C.E. Bear)

Tools for drug development

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In-vitro-Protein based assays:
The Christine Bear lab has also developed novel methods to elucidate how small molecule leads- discovered by other CF research groups act to repair mutant CFTR proteins (G551D-CFTR and ∆F508-CFTR)- assays which also enable design and optimization of the most effective and specific drugs.

In-vivo-Preclinical trials in mice:
Established facilities for preclinical trials in CF Mice (Cftr knockout and ΔF508) (Catherine Luk and Christine Bear)

Preclinical trials in Bronchial airway cells cultured from CF patient lung transplants:
Collaboration with University of Iowa- providing airway cells from CF lung transplants for trials. (Herman Yeger and Neil Sweezey)

Development of clinical tools

Clinical assays:
Drs. Peter Durie and Felix Ratjen have developed new clinical assays which show the promise of enhanced sensitivity and reproducibility – characteristics which are vital for testing the new generation of potential therapies targeting mutant CFTR. (T. Gonska, P. Durie, R. Amin and F. Ratjen)

Skin-based assay:
Dr. Peter Durie has developed a novel, non-invasive and sensitive skin-based assay of the CFTR defect.

Lung-function assay:
Dr. Ratjen is already testing the efficacy and sensitivity of a lung function assay (the lung clearance index) to assess improvement in lung function in two therapy trials.

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