Article

Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway models

2024-06-11

Abstract excerpt

<title>Abstract</title> <p>Mutation-agnostic treatments such as airway gene therapy have the potential to treat any individual with cystic fibrosis (CF), irrespective of their CF transmembrane conductance regulator (<italic>CFTR</italic>) gene variants. The aim of this study was to employ two CF rat models, Phe508del and <italic>CFTR</italic> knockout (KO), to assess the comparative effectiveness of CFTR modulato...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 13fd6bbc-6082-5c79-ac9f-9c15bf07c2df
DOI: 10.21203/rs.3.rs-4470260/v1

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway modelsDOI 10.21203/rs.3.rs-4470260/v1Read

Moving forward: cystic fibrosis gene therapySemantic proximity Read

Current landscape of cystic fibrosis gene therapySemantic proximity Read

Gene-Corrected Basal Cells Restore CFTR In Vitro; Transplants Regenerate Epithelium in a Preclinical Sinus ModelSemantic proximity Read

Advances in gene therapy for cystic fibrosis lung disease.Semantic proximity Read

Cystic fibrosis gene therapy: successes, failures and hopes for the futureSemantic proximity Read

Gene therapy for the treatment of cystic fibrosisSemantic proximity Read

Cystic Fibrosis Gene Therapy: Looking Back, Looking ForwardSemantic proximity Read

Genetic therapies for cystic fibrosis lung diseaseSemantic proximity Read

Advances in Cell and Gene-based Therapies for Cystic Fibrosis Lung DiseaseSemantic proximity Read

Non-viral mRNA cystic fibrosis therapies and their ongoing clinical trials.Semantic proximity Read

Lentiviral Gene Therapy for Cystic Fibrosis: A Promising Approach and First-in-Human Trial.Semantic proximity Read

Cystic fibrosis gene therapy: a mutation-independent treatment.Semantic proximity Read