Publication

Targeted Gene Insertion for Functional CFTR Restoration in Airway Epithelium

Downloadable Content

Persistent URL
Last modified
  • 05/22/2025
Type of Material
Authors
    Cristina Barilla, University of Texas HoustonShingo Suzuki, University of Texas HoustonAndras Rab, Emory UniversityEric Sorscher, Emory UniversityBrian R. Davis, University of Texas Houston
Language
  • English
Date
  • 2022-03-07
Publisher
  • Frontiers Media
Publication Version
Copyright Statement
  • © 2022 Barillà, Suzuki, Rab, Sorscher and Davis.
License
Final Published Version (URL)
Title of Journal or Parent Work
Volume
  • 4
Grant/Funding Information
  • Cystic Fibrosis Foundation: DAVIS17XX0, DAVIS19XX0, DAVIS19XX1, DAVIS20XX2, and SUZUKI19XX0. National Institutes of Health: Animal Models Core of the Center for Gene Therapy of Cystic Fibrosis (P30 DK054759-22) to J. F. Engelhardt.
Abstract
  • Cystic Fibrosis (CF) is caused by a diverse set of mutations distributed across the approximately 250 thousand base pairs of the CFTR gene locus, of which at least 382 are disease-causing (CFTR2.org). Although a variety of editing tools are now available for correction of individual mutations, a strong justification can be made for a more universal gene insertion approach, in principle capable of correcting virtually all CFTR mutations. Provided that such a methodology is capable of efficiently correcting relevant stem cells of the airway epithelium, this could potentially provide life-long correction for the lung. In this Perspective we highlight several requirements for efficient gene insertion into airway epithelial stem cells. In addition, we focus on specific features of the transgene construct and the endogenous CFTR locus that influence whether the inserted gene sequences will give rise to robust and physiologically relevant levels of CFTR function in airway epithelium. Finally, we consider how in vitro gene insertion methodologies may be adapted for direct in vivo editing.
Author Notes
Keywords
Research Categories
  • Health Sciences, Medicine and Surgery
  • Biology, Genetics

Tools

Relations

In Collection:

Items

Thumbnail Title File Description Date Uploaded Visibility Actions
file details Publication File - vvwcs.pdf Primary Content 2025-05-07 Public Download