Turning up the Heat with Therapeutic Epigenome Editing

Alexandra Zezulin; Kiran Musunuru

doi:10.1016/j.stem.2017.12.013

Turning up the Heat with Therapeutic Epigenome Editing

Cell Stem Cell. 2018 Jan 4;22(1):10-11. doi: 10.1016/j.stem.2017.12.013.

Authors

Alexandra Zezulin¹, Kiran Musunuru²

Affiliations

¹ Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
² Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA. Electronic address: kiranmusunuru@gmail.com.

PMID: 29304336
DOI: 10.1016/j.stem.2017.12.013

Abstract

CRISPR-Cas9 has been touted for therapeutic genome editing, but limitations include inefficient correction of disease-causing mutations and off-target mutagenesis. In the latest issue of Cell, Liao et al. (2017) show that a modified version of CRISPR-Cas9 can target and activate key therapeutic genes in vivo without altering DNA sequence identity.

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MeSH terms

Base Sequence
CRISPR-Cas Systems*
Clustered Regularly Interspaced Short Palindromic Repeats
Gene Editing
Mutagenesis*