A non-viral genome editing platform for site-specific insertion of large transgenes.

ABSTRACT:

Background

The precise, functional and safe insertion of large DNA payloads into host genomes offers versatility in downstream genetic engineering-associated applications, spanning cell and gene therapies, therapeutic protein production, high-throughput cell-based drug screening and reporter cell lines amongst others. Employing viral- and non-viral-based genome engineering tools to achieve specific insertion of large DNA-despite being successful in E. coli and animal models-still pose challenges in the human system. In this study, we demonstrate the applicability of our lambda integrase-based genome insertion tool for human cell and gene therapy applications that require insertions of large functional genes, as exemplified by the integration of a functional copy of the F8 gene and

SUBMITTER: Chaudhari N 

PROVIDER: S-EPMC7650303 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature