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The Promise and Challenge of In Vivo Delivery for Genome Therapeutics.

Abstract

CRISPR-based genome editing technologies are poised to enable countless new therapies to prevent, treat, or cure diseases with a genetic basis. However, the safe and effective delivery of genome editing enzymes represents a substantial challenge that must be tackled to enable the next generation of genetic therapies. In this Review, we summarize recent progress in developing enzymatic tools to combat genetic disease and examine current efforts to deliver these enzymes to the cells in need of correction. Viral vectors already in use for traditional gene therapy are being applied to enable in vivo CRISPR-based therapeutics, as are emerging technologies such as nanoparticle-based delivery of CRISPR components and direct delivery of preassembled RNA-protein complexes. Success in these areas will allow CRISPR-based genome editing therapeutics to reach their full potential.

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  • Publisher Full Text
  • Authors+Show Affiliations

    ,

    Innovative Genomics Institute, University of California , Berkeley, California 94720, United States. California Institute for Quantitative Biosciences, University of California , Berkeley, California 94720, United States.

    Innovative Genomics Institute, University of California , Berkeley, California 94720, United States. Helen Diller Family Comprehensive Cancer Center, University of California , San Francisco, California 94158, United States. Department of Urology, University of California , San Francisco, California 94158, United States.

    Source

    ACS chemical biology 13:2 2018 Feb 16 pg 376-382

    Pub Type(s)

    Journal Article

    Language

    eng

    PubMed ID

    29019396