ReviewJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2019
The CF Canada-Sick Kids Program in individual CF therapy: A resource for the advancement of personalized medicine in CF.
Review in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers.
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Who cites it
31 citing papers in PubMed, 62 citations in OpenAlex.
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- The role of GABA in type 1 diabetes.Frontiers in endocrinology · 2024Review
- Theratyping of the Rare CFTR Genotype A559T in Rectal Organoids and Nasal Cells Reveals a Relevant Response to Elexacaftor (VX-445) and Tezacaftor (VX-661) Combination.International journal of molecular sciences · 2023Article
- A Cell-Based Optimised Approach for Rapid and Efficient Gene Editing of Human Pluripotent Stem Cells.International journal of molecular sciences · 2023Article
- Validating organoid-derived human intestinal monolayers for personalized therapy in cystic fibrosis.Life science alliance · 2023Article
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- A flexible summary statistics-based colocalization method with application to the mucin cystic fibrosis lung disease modifier locus.American journal of human genetics · 2022Article
- Insulin-Like Growth Factor Binding Protein (IGFBP-6) as a Novel Regulator of Inflammatory Response in Cystic Fibrosis Airway Cells.Frontiers in molecular biosciences · 2022Article
- "The Stakes Are Higher"- Patient and Caregiver Perspectives on Cystic Fibrosis Research and Personalized Medicine.Frontiers in medicine · 2022Article
- Article
- Generation of functional ciliated cholangiocytes from human pluripotent stem cells.Nature communications · 2021Article
- A new platform for high-throughput therapy testing on iPSC-derived lung progenitor cells from cystic fibrosis patients.Stem cell reports · 2021Article
- Perspectives on the translation of in-vitro studies to precision medicine in Cystic Fibrosis.EBioMedicine · 2021Review
- Gene therapy for cystic fibrosis: new tools for precision medicine.Journal of translational medicine · 2021Review
- Elexacaftor co-potentiates the activity of F508del and gating mutants of CFTR.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2021Article
- Inflammatory epithelial cytokines afterERJ open research · 2021Article
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Authors and funding
24 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundTherapies targeting certain CFTR mutants have been approved, yet variations in clinical response highlight the need for in-vitro and genetic tools that predict patient-specific clinical outcomes. Toward this goal, the CF Canada-Sick Kids Program in Individual CF Therapy (CFIT) is generating a "first of its kind", comprehensive resource containing patient-specific cell cultures and data from 100 CF individuals that will enable modeling of therapeutic responses.
methodsThe CFIT program is generating: 1) nasal cells from drug naïve patients suitable for culture and the study of drug responses in vitro, 2) matched gene expression data obtained by sequencing the RNA from the primary nasal tissue, 3) whole genome sequencing of blood derived DNA from each of the 100 participants, 4) induced pluripotent stem cells (iPSCs) generated from each participant's blood sample, 5) CRISPR-edited isogenic control iPSC lines and 6) prospective clinical data from patients treated with CF modulators.
resultsTo date, we have recruited 57 of 100 individuals to CFIT, most of whom are homozygous for F508del (to assess in-vitro: in-vivo correlations with respect to ORKAMBI response) or heterozygous for F508del and a minimal function mutation. In addition, several donors are homozygous for rare nonsense and missense mutations. Nasal epithelial cell cultures and matched iPSC lines are available for many of these donors.
conclusionsThis accessible resource will enable development of tools that predict individual outcomes to current and emerging modulators targeting F508del-CFTR and facilitate therapy discovery for rare CF causing mutations.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.