Cystic Fibrosis

CT ASSESSMENT OF BODY COMPOSITION AND LUNG EVOLUTION IN CYSTIC FIBROSIS AFTER ELEXACAFTOR-TEZACAFTOR-IVACAFTOR: A COHORT STUDY.

BACKGROUND: Elexacaftor-Tezacaftor-Ivacaftor (ETI) therapy markedly improves pulmonary function in people with cystic fibrosis (pwCF) but induces weight gain, raising concerns about body composition. CT scans routinely performed to assess lung structure offer opportunistic nutritional assessment. In this study, we aimed to evaluate changes in body composition in pwCF after ETI through CT image ana...

Decoding the role of H19 in cholestatic liver injury using snRNA-seq, spatial transcriptomics, and machine learning-based disease prediction.

BACKGROUND: Despite recent advances, Primary Sclerosing Cholangitis (PSC)-a chronic obstructive biliary disease-still lacks effective therapies to prevent disease progression or the need for liver transplantation. Moreover, up to 30% of transplant recipients experience recurrence. Long non-coding RNA H19 (H19) has been implicated in promoting PSC progression, yet the cellular and molecular mechani...

An Overlooked Formulation Variable: Solvent Reshapes DNA Delivery.

Nonviral polymeric vectors offer a tunable platform for nucleic acid delivery, yet formulation variables beyond polymer structure remain underexplored...

CFTR modulator therapy reshapes airway inflammation in cystic fibrosis: Insights from proteomics and machine learning.

BACKGROUND: CFTR modulators, including Elexacaftor/Tezacaftor/Ivacaftor (ETI), have markedly improved clinical outcomes for people with cystic fibrosi...

The regional landscape of the human colon culturome in health and cystic fibrosis.

UNLABELLED: Cystic fibrosis (CF) alters gut physiology, yet its impact on microbial communities across colonic regions (ascending, transverse, descend...

3D Automated Segmentation of Bronchial Abnormalities on Ultrashort Echo Time MRI: A Quantitative MR Outcome in Cystic Fibrosis.

BACKGROUND: Cystic fibrosis (CF) monitoring relies on computed tomography (CT), but ultra-short echo time MRI (UTE-MRI) offers a radiation-free altern...

Identification of structural predictors of lung function improvement in adults with cystic fibrosis treated with elexacaftor-tezacaftor-ivacaftor using deep-learning.

PURPOSE: The purpose of this study was to evaluate the relationship between structural abnormalities on CT and lung function prior to and after initia...

Protocol for functional screening of CFTR-targeted genetic therapies in patient-derived organoids using DETECTOR deep-learning-based analysis.

Here, we present a protocol for the rapid functional screening of gene editing and addition strategies in patient-derived organoids using the deep-lea...

Artificial Intelligence-Driven Prediction Revealed CFTR Associated with Therapy Outcome of Breast Cancer: A Feasibility Study.

INTRODUCTION: In silico tools capable of predicting the functional consequences of genomic differences between individuals, many of which are AI-drive...

OrgaSegment: deep-learning based organoid segmentation to quantify CFTR dependent fluid secretion.

Epithelial ion and fluid transport studies in patient-derived organoids (PDOs) are increasingly being used for preclinical studies, drug development a...

Systematical analysis of underlying markers associated with Marfan syndrome via integrated bioinformatics and machine learning strategies.

Marfan syndrome (MFS) is a hereditary disease with high mortality. This study aimed to explore peripheral blood potential markers and underlying mecha...

Sweat Proteomics in Cystic Fibrosis: Discovering Companion Biomarkers for Precision Medicine and Therapeutic Development.

In clinical routine, the diagnosis of cystic fibrosis (CF) is still challenging regardless of international consensus on diagnosis guidelines and test...

Artificial intelligence in computed tomography for quantifying lung changes in the era of CFTR modulators.

BACKGROUND: Chest computed tomography (CT) remains the imaging standard for demonstrating cystic fibrosis (CF) airway structural disease . However, vi...

A deep learning approach to identify gene targets of a therapeutic for human splicing disorders.

Pre-mRNA splicing is a key controller of human gene expression. Disturbances in splicing due to mutation lead to dysregulated protein expression and c...

Deep-learning based repurposing of FDA-approved drugs against dihydrofolate reductase and molecular dynamics study.

causes the fatal fungal bloodstream infection in humans called Candidiasis. Most of the species are resistant to the antifungals used to treat them....

Cyst-type epithelial heterogeneity shapes therapeutic responsiveness in ADPKD

Autosomal dominant polycystic kidney disease (ADPKD) exhibits substantial interpatient variability in disease course and therapeutic response, but the...

Host Microenvironment Reprogramming by Saccharides Overcomes Lung Barriers for mRNA Therapeutics

Overcoming biological barriers remains the paramount challenge for pulmonary mRNA therapeutics. Conventional approaches focus exclusively on passively...

The Regional Landscape of the Human Colon Culturome in Health and Cystic Fibrosis

Cystic fibrosis (CF) alters gut physiology, yet its impact on microbial communities across colonic regions (ascending, transverse, descending colon) a...

Serum Exosomal Multi-Omic Signatures Stratify Glucose Tolerance in Cystic Fibrosis and Reveal Partial Therapeutic Reprogramming by CFTR Modulators

Cystic fibrosis-related diabetes (CFRD) affects up to 60% of adults with CF and contributes to poorer clinical outcomes, including accelerated lung de...

Impact of Cystic Fibrosis Transmembrane Conductance Regulator Therapy on Chronic Rhinosinusitis and Health Status: Deep Learning CT Analysis and Patient-reported Outcomes.

Elexacaftor, tezacaftor, and ivacaftor (ETI) in triple combination improves pulmonary health for people with cystic fibrosis (PwCF). However, its imp...