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Adenoviral vectors for in vivo delivery of CRISPR-Cas gene editors.

ABSTRACT: Harnessing the bacterial clustered regularly interspaced short palindromic repeats (CRISPR) system for genome editing in eukaryotes has revolutionized basic biomedical research and translational sciences. The ability to create targeted alterations of the genome through this easy to design system has presented unprecedented opportunities to treat inherited disorders and other diseases such as cancer through gene therapy. A major hurdle is the lack of an efficient and safe in vivo delivery system, limiting most of the current gene therapy efforts to ex vivo editing of extracted cells. Here we discuss the unique features of adenoviral vectors that enable tissue specific and efficient delivery of the CRISPR-Cas machinery for in vivo genome editing.

SUBMITTER: Boucher P

PROVIDER: S-EPMC8091654 | biostudies-literature | 2020 Nov

REPOSITORIES: biostudies-literature

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Adenoviral vectors for in vivo delivery of CRISPR-Cas gene editors.

Boucher Paul P Cui Xiaoxia X Curiel David T DT

Journal of controlled release : official journal of the Controlled Release Society 20200903

Harnessing the bacterial clustered regularly interspaced short palindromic repeats (CRISPR) system for genome editing in eukaryotes has revolutionized basic biomedical research and translational sciences. The ability to create targeted alterations of the genome through this easy to design system has presented unprecedented opportunities to treat inherited disorders and other diseases such as cancer through gene therapy. A major hurdle is the lack of an efficient and safe in vivo delivery system, ...[more]

PMID: 32891680

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