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Trends Mol Med. 2018 Oct;24(10):822-824. doi: 10.1016/j.molmed.2018.07.008. Epub 2018 Aug 10.

Emerging Strategies for Genome Editing in the Brain.

Author information

1
Innovative Genomics Institute, University of California, Berkeley, California 94720, United States; California Institute for Quantitative Biosciences, University of California, Berkeley, California 94720, United States; ORCID https://orcid.org/0000-0003-2937-4703.
2
Innovative Genomics Institute, University of California, Berkeley, California 94720, United States; California Institute for Quantitative Biosciences, University of California, Berkeley, California 94720, United States; ORCID https://orcid.org/0000-0002-0644-5540. Electronic address: rosswilson@berkeley.edu.

Abstract

Despite the unparalleled therapeutic promise of genome editing, its curative power is currently limited by the substantial difficulty in delivering DNA-cutting enzymes to the cells in need of correction. A recent study demonstrates the potential for the delivery of pre-assembled genome-editing enzymes in the form of ribonucleoprotein complexes, which were used to rescue a mouse model of fragile X syndrome (FXS).

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