ReviewJournal of translational medicine2017
Cystic fibrosis: current therapeutic targets and future approaches.
Review in Journal of translational medicine, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 101 papers, 6 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
101 citing papers in PubMed, 6 syntheses or guidelines pooled it.
- A systematic review and meta-analysis of the treatment modalities available for children afflicted from cystic fibrosis.BMC pediatrics · 2025Pooled it
- Impact of Gene Modifiers on Cystic Fibrosis Phenotypic Profiles: A Systematic Review.Human mutation · 2024Pooled it
- Surgery for nasal polyposis in cystic fibrosis.The Cochrane database of systematic reviews · 2023Pooled it
- Bisphosphonates for osteoporosis in people with cystic fibrosis.The Cochrane database of systematic reviews · 2023Pooled it
- Assessing the consistency of iPSC and animal models in cystic fibrosis modelling: A meta-analysis.PloS one · 2022Pooled it
- Effectiveness of positive expiratory pressure on patients over 16 years of age with cystic fibrosis: systematic review and meta-analysis.Therapeutic advances in respiratory diseasePooled it
- Balancing hope and uncertainty: family perspectives on lung transplantation in cystic fibrosis - a qualitative study.International journal of qualitative studies on health and well-being · 2026Article
- A DNA Tetrahedron-Delivered miR-17-5p mimic exerts dual anti-inflammatory and Anti-Non-coding RNA research · 2026Article
- Combining Gene Therapy with Current Modulator Treatments for Cystic Fibrosis: A Promising Area of Research.Pharmaceutics · 2026Review
- Impact of Elexacaftor/Tezacaftor/Ivacaftor on Systemic Antibiotic Utilization in Pediatric Patients With Cystic Fibrosis.Pediatric pulmonology · 2026Article
- Review
- "I know that exercise is very good for me": perception of pediatric patients with cystic fibrosis about physical exercise program.Revista paulista de pediatria : orgao oficial da Sociedade de Pediatria de Sao Paulo · 2026Article
- Hidden connection: Impact of the gut microbiota on respiratory diseases in children (Review).Molecular medicine reports · 2026Review
- The role of Na,K‑ATPase in lung diseases (Review).Molecular medicine reports · 2025Review
- Cystic Fibrosis and CFTR Modulators: The Impact on Bone Density, Muscle Mass and Strength in Children and Young Adolescents.Children (Basel, Switzerland) · 2025Review
- The Epidemiology of Nontuberculous Mycobacteria in Cystic Fibrosis.Children (Basel, Switzerland) · 2025Review
- Airway Microbiome in Children with Cystic Fibrosis: A Review of Microbial Shifts and Therapeutic Impacts.Medicina (Kaunas, Lithuania) · 2025Review
- Optimizing Reproductive Autonomy in Cystic Fibrosis: The Role of Oocyte Cryopreservation.Journal of reproduction & infertility · 2025Article
- Ceftobiprole in cystic fibrosis: a case series.JAC-antimicrobial resistance · 2025Article
- Innate Immunity in Cystic Fibrosis: Varied Effects of CFTR Modulator Therapy on Cell-to-Cell Communication.International journal of molecular sciences · 2025Review
41 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectivesStudy of currently approved drugs and exploration of future clinical development pipeline therapeutics for cystic fibrosis, and possible limitations in their use.
methodsExtensive literature search using individual and a combination of key words related to cystic fibrosis therapeutics. KEY
findingsCystic fibrosis is an autosomal recessive disorder due to mutations in CFTR gene leading to abnormality of chloride channels in mucus and sweat producing cells. Respiratory system and GIT are primarily involved but eventually multiple organs are affected leading to life threatening complications. Management requires drug therapy, extensive physiotherapy and nutritional support. Previously, the focus was on symptomatic improvement and complication prevention but recently the protein rectifiers are being studied which are claimed to correct underlying structural and functional abnormalities. Some improvement is observed by the corrector drugs. Other promising approaches are gene therapy, targeting of cellular interactomes, and newer drugs for symptomatic improvement.
conclusionsThe treatment has a long way to go as most of the existing therapeutics is for older children. Other limiting factors include mutation class, genetic profile, drug interactions, adverse effects, and cost. Novel approaches like gene transfer/gene editing, disease modeling and search for alternative targets are warranted.
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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.