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

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

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...

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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.
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