@inbook{8a073f1a900a4e52b37fb1cba4aeec34,
title = "Targeted therapeutic genome engineering: Opportunities and bottlenecks in medical translation",
abstract = "Tools for editing the genome and epigenome represent a new frontier in targeted therapeutic intervention. Nonviral nanoparticle-based delivery systems are potentially the long-term future for delivering gene therapies in vivo in a safe and controllable manner. Currently, nonviral agents fail to couple delivery efficiency across cell type with low toxicity and the ability to deliver large or multiple payloads, rendering clinical implementation of these technologies elusive. Herein we review recent developments in the evaluation of polymeric and inorganic nanoparticles as nonviral methods to deliver current genome editing tools. In particular, this chapter will focus on in vivo delivery of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and the CRISPR-associated protein 9 (Cas9), and the current bottlenecks the field faces.",
author = "Kretzmann, {Jessica A.} and Luther, {David C.} and Marck Norret and Rotello, {Vincent M.} and Iyer, {K. Swaminathan}",
year = "2019",
month = mar,
day = "20",
doi = "10.1021/bk-2019-1309.ch001",
language = "English",
isbn = "9780841233836",
series = "ACS Symposium Series",
publisher = "Rubber Division of the American Chemical Society",
pages = "1--34",
editor = "Ilies, {Marc A.} and Kazuo Sakurai",
booktitle = "Targeted Nanosystems for Therapeutic Applications",
address = "United States",
}