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Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics.

ABSTRACT: The recent progress in harnessing the efficient and precise method of DNA editing provided by CRISPR/Cas9 is one of the most promising major advances in the field of gene therapy. However, the development of safe and optimally efficient delivery systems for CRISPR/Cas9 elements capable of achieving specific targeting of gene therapy to the location of interest without off-target effects is a primary challenge for clinical therapeutics. Nanoparticles (NPs) provide a promising means to meet such challenges. In this review, we present the most recent advances in developing innovative NP-based delivery systems that efficiently deliver CRISPR/Cas9 constructs and maximize their effectiveness.

SUBMITTER: Givens BE

PROVIDER: S-EPMC6398936 | biostudies-literature | 2018 Oct

REPOSITORIES: biostudies-literature

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Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics.

Givens Brittany E BE Naguib Youssef W YW Geary Sean M SM Devor Eric J EJ Salem Aliasger K AK

The AAPS journal 20181010 6

The recent progress in harnessing the efficient and precise method of DNA editing provided by CRISPR/Cas9 is one of the most promising major advances in the field of gene therapy. However, the development of safe and optimally efficient delivery systems for CRISPR/Cas9 elements capable of achieving specific targeting of gene therapy to the location of interest without off-target effects is a primary challenge for clinical therapeutics. Nanoparticles (NPs) provide a promising means to meet such c ...[more]

PMID: 30306365

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