Journal
CELL SYSTEMS
Volume 10, Issue 1, Pages 1-14Publisher
CELL PRESS
DOI: 10.1016/j.cels.2019.10.003
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Funding
- NIH-NIBIB [R01 EB02456]
- NIH-NHLBI [R01HL141805]
- Arizona Biomedical Research Council New Investigator Award [ADHS16-162402]
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Pinpoint control over endogenous gene expression in vivo has long been a fevered dream for clinicians and researchers alike. With the recent repurposing of programmable, RNA-guided DNA endonucleases from the CRISPR bacterial immune system, this dream is becoming a powerful reality. Engineered CRISPR/Cas9-based transcriptional regulators and epigenome editors have enabled researchers to perturb endogenous gene expression in vivo, allowing for the therapeutic reprogramming of cell and tissue behavior. For this technology to be of maximal use, a variety of technological hurdles still need to be addressed. Better understanding of the design principle controlling gene expression together with technologies that enable spatiotemporal control of transcriptional engineering are fundamental for rational design, improved efficacy, and ultimately safe translation to humans. In this review, we will discuss recent advances and integrative strategies that can help pave the path toward a new class of transcriptional therapeutics.
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