Investigating Adverse Genomic and Regulatory Changes Caused by Replacement of the Full-Length CFTR Cdna Using Cas9 and AAV.

Sriram Vaidyanathan,Jenny L. Kerschner,Alekh Paranjapye, Vrishti Sinha,Brian Lin,Tracy A. Bedrosian,Adrian J. Thrasher,Giandomenico Turchiano,Ann Harris,Matthew H. Porteus

MOLECULAR THERAPY NUCLEIC ACIDS（2024）

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MT: RNA/DNA Editing,cystic fibrosis,CFTR super-exon,CFTR gene regulation,airway basal cell differentiation,genetic rearrangements,airway stem cell therapy,CFTR genome editing,universal CFTR correction

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