Targeted therapeutic genome engineering: Opportunities and bottlenecks in medical translation

Jessica A. Kretzmann, David C. Luther, Marck Norret, Vincent M. Rotello, K. Swaminathan Iyer

Research output: Chapter in Book/Conference paperChapterpeer-review

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.

Original languageEnglish
Title of host publicationTargeted Nanosystems for Therapeutic Applications
Subtitle of host publicationNew Concepts, Dynamic Properties, Efficiency, and Toxicity
EditorsMarc A. Ilies, Kazuo Sakurai
Place of PublicationWashington, DC
PublisherRubber Division of the American Chemical Society
Chapter1
Pages1-34
Number of pages34
ISBN (Electronic)9780841233744
ISBN (Print)9780841233836
DOIs
Publication statusPublished - 20 Mar 2019

Publication series

NameACS Symposium Series
Volume1309
ISSN (Print)0097-6156
ISSN (Electronic)1947-5918

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