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Potential of helper-dependent Adenoviral vectors in CRISPR-cas9-mediated lung gene therapy

23 Jul 2021

Abstract excerpt

Since CRISPR/Cas9 was harnessed to edit DNA, the field of gene therapy has witnessed great advances in gene editing. New avenues were created for the treatment of diseases such as Cystic Fibrosis (CF). CF is caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Despite the success of gene editing with the CRISPR/Cas9 in vitro, challenges still exist when using CRISPR/Cas9 in...

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PMID: 34301308
DOI: 10.1186/s13578-021-00662-w

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