Evidence map›Paper›PMID 40282362›Full record

ReviewGenes2025

Cutting-Edge Advances in Cystic Fibrosis: From Gene Therapy to Personalized Medicine and Holistic Management.

Giuseppe Fabio Parisi, Vito Terlizzi, Sara Manti, Maria Papale, Giulia Pecora, Santiago Presti, Monica Tosto, Salvatore Leonardi

Abstract readReview
In one paragraph

Review in Genes, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

6 citing papers in PubMed.

  1. Review
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors.

Giuseppe Fabio ParisiPediatric Respiratory Unit, Department of Clinical and Experimental Medicine, San Marco Hospital, University of Catania, 95121 Catania, Italy.ORCID 0000-0003-4291-0195
Vito TerlizziCystic Fibrosis Regional Reference Centre, Department of Paediatric Medicine, Meyer Children's Hospital IRCCS, Viale Gaetano Pieraccini 24, 50139 Florence, Italy.ORCID 0000-0003-1106-4424
Sara MantiPediatric Unit, Department of Human and Pediatric Pathology "Gaetano Barresi", Azienda Ospedaliera Universitaria Policlinico "G. Martino", University of Messina, Via Consolare Valeria, 1, 98124 Messina, Italy.ORCID 0000-0001-5420-8411
Maria PapalePediatric Respiratory Unit, Department of Clinical and Experimental Medicine, San Marco Hospital, University of Catania, 95121 Catania, Italy.
Giulia PecoraPediatric Respiratory Unit, Department of Clinical and Experimental Medicine, San Marco Hospital, University of Catania, 95121 Catania, Italy.ORCID 0009-0008-3381-9844
Santiago PrestiPediatric Respiratory Unit, Department of Clinical and Experimental Medicine, San Marco Hospital, University of Catania, 95121 Catania, Italy.ORCID 0000-0002-4879-5418
Monica TostoPediatric Respiratory Unit, Department of Clinical and Experimental Medicine, San Marco Hospital, University of Catania, 95121 Catania, Italy.
Salvatore LeonardiPediatric Respiratory Unit, Department of Clinical and Experimental Medicine, San Marco Hospital, University of Catania, 95121 Catania, Italy.ORCID 0000-0002-2642-6400

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Cystic fibrosis (CF), a genetic disorder characterized by mutations in the CFTR gene, has seen significant advances in treatment through cutting-edge approaches such as gene therapy and personalized medicine. This review examines the current and emerging strategies shaping CF care, focusing on novel therapies that target the root cause of CF and optimize patient outcomes. CFTR modulators have transformed cystic fibrosis management by enhancing protein function for specific mutations, leading to improved lung function and quality of life. Concurrently, gene therapy offers transformative potential by aiming to correct CFTR mutations using tools like CRISPR/Cas9 or prime editing, though challenges remain in delivery and long-term efficacy. The integration of precision medicine, facilitated by genomic and computational technologies, allows for personalized treatment plans that account for genetic variability and disease severity. Complementing these approaches, holistic management emphasizes the importance of psychological support and nutritional optimization, acknowledging CF's multi-system impact. Future directions include exploring anti-inflammatory agents and microbiome modulation to further mitigate disease morbidity. However, global disparities in treatment access continue to challenge equitable healthcare delivery, underscoring the need for policy reform and international cooperation. By synthesizing these developments, this review highlights the transformative potential of modern CF treatments, advocating for continued innovation and global healthcare equity, with the ultimate goal of dramatically improving life expectancy and quality of life for individuals with CF.

Indexed as

Cystic FibrosisGenetic TherapyPrecision MedicineCystic Fibrosis Transmembrane Conductance RegulatorGene EditingHolistic HealthHumansMutationQuality of LifeCFTR protein, humanCystic Fibrosis Transmembrane Conductance RegulatorCFTRcystic fibrosisgene therapymodulatorspersonalized therapy

Identifiers

PMID40282362
PMCPMC12026723

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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.