Treating human genetic conditions in vivo requires efficient delivery of the CRISPR gene editing machinery to the affected cells and organs. The gene editing field has seen clinical advances with ex vivo therapies and with in vivo delivery to the liver using lipid nanoparticle technology. Adeno-associated virus (AAV) serotypes have been discovered and engineered to deliver genetic material to nearly every organ in the body. However, the large size of most CRISPR-Cas systems limits packaging into the viral genome and reduces drug development flexibility and manufacturing efficiency. Here, we demonstrate efficient CRISPR gene editing using a miniature CRISPR-Cas12f system with expanded genome targeting packaged into AAV particles. We identified efficient guides for four therapeutic gene targets and encoded the guides and the Cas12f nuclease into a single AAV. We then demonstrate editing in multiple cell lines, patient fibroblasts, and primary hepatocytes. We then screened the cells for off-target editing, demonstrating the safety of the therapeutics. These results represent an important step in applying CRISPR editing to diverse genetic sequences and organs in the body.

Allison Sharrar

Zuriah Meacham

Johanna Staples-Ager

Luisa Arake de Tacca

David Rabuka

Trevor Collingwood

Michael Schelle

The CRISPR Journal

为表彰这一非凡的科技时代，MARY ANN LIEBERT, INC. 的出版商最近宣布创建 THE CRISPR JOURNAL——这是一份国际性、多学科的同行评审期刊，发表关于 CRISPR 无数应用和基础技术的杰出研究。 2018 年，CRISPR 期刊将以灵活的开放获取选项在线和印刷出版，为开创性研究以及生动和挑衅性的评论、分析和辩论提供一个备受瞩目的场所。 CRISPR 期刊为 MARY ANN LIEBERT, INC. 的产品组合增加了一个令人兴奋和充满活力的组成部分，其中包括 GEN（基因工程和生物技术新闻）和 80 多个领先的同行评审期刊。

To celebrate this extraordinary era of technology, the publisher MARY ANN LIEBERT, INC. recently announced the creation of The CRISPR Journal. This is an international, multidisciplinary peer-reviewed journal dedicated to publishing outstanding research on the numerous applications and foundational technologies of CRISPR. Starting in 2018, The CRISPR Journal will be published online and in print with flexible open access options, providing a prominent venue for groundbreaking research as well as vibrant and provocative commentary, analysis, and debate. The CRISPR Journal adds an exciting and dynamic component to the portfolio of MARY ANN LIEBERT, INC., which includes GEN (Genetic Engineering and Biotechnology News) and over 80 leading peer-reviewed journals.

为了庆祝这一非凡的科技时代，MARY ANN LIEBERT, INC. 的出版商最近宣布创办《CRISPR Journal》。这是一份国际性、多学科的同行评审期刊，专注于发表关于CRISPR技术及其众多应用的杰出研究。自2018年起，《CRISPR Journal》将以灵活的开放获取选项在线和印刷出版，为开创性研究以及生动且富有挑战性的评论、分析和辩论提供一个备受瞩目的平台。《CRISPR Journal》为MARY ANN LIEBERT, INC.的产品组合增添了一个令人兴奋和充满活力的组成部分，涵盖了GEN（基因工程与生物技术新闻）及80多个领先的同行评审期刊。

Category	Quartile
生物学	4
生物学, 遗传学	4

Viral Delivery of Compact CRISPR-Cas12f for Gene Editing Applications

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Journal Info

Category	Quartile
GENETICS & HEREDITY	2