ReviewHuman gene therapy2015
Cystic Fibrosis Gene Therapy in the UK and Elsewhere.
Review in Human gene therapy, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 42 papers.
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
42 citing papers in PubMed.
- Development of CpG-Depleted CFTR Plasmid-Based Nanoparticles for Nonviral Gene Therapy in Lung Cystic Fibrosis Disease.The journal of gene medicine · 2026Article
- AAV-based gene delivery of antimicrobial peptides to combat drug-resistant pathogens.Applied and environmental microbiology · 2025Review
- The Road Less Traveled: Slow but Steady Progress Toward Cystic Fibrosis Gene Therapy by the UK Respiratory Gene Therapy Consortium.American journal of respiratory and critical care medicine · 2024Article
- DNA-PKcs inhibition improves sequential gene insertion of the full-lengthMolecular therapy. Nucleic acids · 2024Article
- Highly branched poly β-amino ester/CpG-depletedMolecular therapy. Methods & clinical development · 2024Article
- DNA-PKcs Inhibition Improves Sequential Gene Insertion of the Full-LengthbioRxiv : the preprint server for biology · 2024Article
- Nanomedicines via the pulmonary route: a promising strategy to reach the target?Drug delivery and translational research · 2024Review
- Compound heterozygous mutations in CFTR causing congenital bilateral absence of the vas deferens in a Chinese pedigree.Molecular genetics & genomic medicine · 2024Article
- Opportunities and Challenges for Inhalable Nanomedicine Formulations in Respiratory Diseases: A Review.International journal of nanomedicine · 2024Review
- Biomaterial-based gene therapy.MedComm · 2023Review
- Gene therapy and gene correction: targets, progress, and challenges for treating human diseases.Gene therapy · 2022Review
- Computational Genomics in the Era of Precision Medicine: Applications to Variant Analysis and Gene Therapy.Journal of personalized medicine · 2022Review
- Gene Therapy Approach with an Emphasis on Growth Factors: Theoretical and Clinical Outcomes in Neurodegenerative Diseases.Molecular neurobiology · 2022Review
- Treatment of Pulmonary Disease of Cystic Fibrosis: A Comprehensive Review.Antibiotics (Basel, Switzerland) · 2021Review
- Targeted Nanotherapeutics for Respiratory Diseases: Cancer, Fibrosis, and Coronavirus.Advanced therapeutics · 2021Review
- Recent applications and strategies in nanotechnology for lung diseases.Nano research · 2021Review
- Treatment of Cystic Fibrosis: From Gene- to Cell-Based Therapies.Frontiers in pharmacology · 2021Review
- New Therapeutic Approaches in Cystic Fibrosis.Turkish journal of pharmaceutical sciences · 2020Article
- Human germline editing in the era of CRISPR-Cas: risk and uncertainty, inter-generational responsibility, therapeutic legitimacy.BMC medical ethics · 2020Article
- Gene Therapy for Respiratory Diseases: Progress and a Changing Context.Human gene therapy · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
Abstract
The cystic fibrosis transmembrane conductance regulator (CFTR) gene was identified in 1989. This opened the door for the development of cystic fibrosis (CF) gene therapy, which has been actively pursued for the last 20 years. Although 26 clinical trials involving approximately 450 patients have been carried out, the vast majority of these trials were short and included small numbers of patients; they were not designed to assess clinical benefit, but to establish safety and proof-of-concept for gene transfer using molecular end points such as the detection of recombinant mRNA or correction of the ion transport defect. The only currently published trial designed and powered to assess clinical efficacy (defined as improvement in lung function) administered AAV2-CFTR to the lungs of patients with CF. The U.K. Cystic Fibrosis Gene Therapy Consortium completed, in the autumn of 2014, the first nonviral gene therapy trial designed to answer whether repeated nonviral gene transfer (12 doses over 12 months) can lead to clinical benefit. The demonstration that the molecular defect in CFTR can be corrected with small-molecule drugs, and the success of gene therapy in other monogenic diseases, is boosting interest in CF gene therapy. Developments are discussed here.
Indexed as
Identifiers
What OpenQuestion holds
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.