Investigating adverse genomic and regulatory changes caused by replacement of the full-length CFTR cDNA using Cas9 and AAV

• Main Authors: Sriram Vaidyanathan (Author), Jenny L. Kerschner (Author), Alekh Paranjapye (Author), Vrishti Sinha (Author), Brian Lin (Author), Tracy A. Bedrosian (Author), Adrian J. Thrasher (Author), Giandomenico Turchiano (Author), Ann Harris (Author), Matthew H. Porteus (Author)
• Format: Book
• Published: Elsevier, 2024-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

A "universal strategy" replacing the full-length CFTR cDNA may treat >99% of people with cystic fibrosis (pwCF), regardless of their specific mutations. Cas9-based gene editing was used to insert the CFTR cDNA and a truncated CD19 (tCD19) enrichment tag at the CFTR locus in airway basal...