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National Institute of Environmental Health Sciences

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Publication Detail

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

Authors: Givens, Brittany E; Naguib, Youssef W; Geary, Sean M; Devor, Eric J; Salem, Aliasger K

Published In AAPS J, (2018 Oct 10)

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.

PubMed ID: 30306365 Exiting the NIEHS site

MeSH Terms: CRISPR-Cas Systems/genetics*; DNA/genetics; Gene Editing/methods*; Gene Transfer Techniques*; Genetic Therapy/methods*; Humans; Nanoparticles/chemistry*

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