Article

A Dual-Locus-Targeting Strategy to Enhance CRISPR/Cas9-mediated CFTR Replacement via Helper-Dependent Adenoviral vector in porcine genome

2026-06-11

Abstract excerpt

<h4>ABSTRACT</h4> Gene therapy has been the subject of extensive research following the advent of gene-editing technologies. Genetic disorders with difficult-to-target tissues, such as cystic fibrosis (CF), still face many challenges in developing efficacious gene therapy. The potential universal approach of gene replacement involves inserting a functional CFTR gene after generating DNA double strand breaks usin...

Topics

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

Identifiers and source

Literature Corpus work: dd8cf914-e6fd-5c07-a920-ef6bc51a4c7c
DOI: 10.64898/2026.06.10.731381

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

A Dual-Locus-Targeting Strategy to Enhance CRISPR/Cas9-mediated CFTR Replacement via Helper-Dependent Adenoviral vector in porcine genomeDOI 10.64898/2026.06.10.731381Read

Potential of helper-dependent Adenoviral vectors in CRISPR-cas9-mediated lung gene therapySemantic proximity Read

In Vitro Validation of a CRISPR-Mediated CFTR Correction Strategy for Preclinical Translation in PigsSemantic proximity Read

Validation of caprine H11 and Rosa26 safe harbor loci via CRISPR- based system: Investigations on stable transgene expression and genetic biosafetySemantic proximity Read

Identification of the safe harbor locus, AAVS1, from porcine genome and site-specific integration of recombinase-mediated cassette exchange system in porcine fibroblasts using CRISPR/Cas9Semantic proximity Read

Innovative Therapeutic Strategies for Cystic Fibrosis: Moving Forward to CRISPR TechniqueSemantic proximity Read

CRISPR/Cas9-mediated integration of large transgene into pig potential safe harborSemantic proximity Read

CRISPR/Cas9 gene editing therapies for cystic fibrosisSemantic proximity Read

Targeted replacement of full-length CFTR in human airway stem cells by CRISPR/Cas9 for pan-mutation correction in the endogenous locusSemantic proximity Read

Gene therapy for cystic fibrosis: Challenges and prospectsSemantic proximity Read

Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus.Semantic proximity Read

Efficient Gene Delivery to Pig Airway Epithelia and Submucosal Glands Using Helper-Dependent Adenoviral VectorsSemantic proximity Read

Enhanced CRISPR/Cas9-mediated biallelic genome targeting with dual surrogate reporter-integrated donors.Semantic proximity Read