Efficient gene editing in adult mouse livers via adenoviral delivery of CRISPR/Cas9
Ranran Cheng,Jin Peng,Yan Yonghong,Peili Cao,Jiewei Wang,Chen Qiu,Lichun Tang,Di Liu,Tang Li,Jianping Jin,Xingxu Huang,Fuchu He,Pumin Zhang +12 more
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TLDR
An adenovirus‐based CRISPR/Cas9 system for gene editing in vivo is developed and demonstrated that the system could reach the level of tissue‐specific gene knockout, resulting in phenotypic changes in the liver.About:
This article is published in FEBS Letters.The article was published on 2014-11-03 and is currently open access. It has received 113 citations till now. The article focuses on the topics: CRISPR & Cas9.read more
Citations
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Journal ArticleDOI
CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes.
Alexis C. Komor,Alexis C. Komor,Alexis C. Komor,Ahmed H. Badran,Ahmed H. Badran,Ahmed H. Badran,David R. Liu,David R. Liu,David R. Liu +8 more
TL;DR: Recent developments that extend the generality, DNA specificity, product selectivity, and fundamental capabilities of natural CRISPR systems are described.
Journal ArticleDOI
Delivering CRISPR: a review of the challenges and approaches.
TL;DR: The focus then turns to the most difficult barrier to potential in vivo use of CRISPR/Cas9, delivery, and detail the various cargos and delivery vehicles reported for CRISpr/ Cas9, including physical delivery vehicles, viral delivery methods, and non-viral delivery methods.
Journal ArticleDOI
A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice
Yang Yang,Lili Wang,Peter Bell,Deirdre McMenamin,Zhenning He,John H. White,Hongwei Yu,Chenyu Xu,Hiroki Morizono,Kiran Musunuru,Mark L. Batshaw,James M. Wilson +11 more
TL;DR: In this paper, a CRISPR-Cas9-based approach was used for gene correction in newborn mice with a partial deficiency in the urea cycle disorder enzyme, ornithine transcarbamylase (OTC).
Journal ArticleDOI
Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses.
Dan Wang,Haiwei Mou,Shaoyong Li,Yingxiang Li,Soren Hough,Karen Tran,Jia Li,Hao Yin,Daniel G. Anderson,Erik J. Sontheimer,Zhiping Weng,Guangping Gao,Wen Xue +12 more
TL;DR: In this article, the authors used an Ad vector to deliver a Streptococcus pyogenes-derived Cas9 system (SpCas9) targeting Pten, a gene involved in NASH and a negative regulator of the PI3K-AKT pathway.
Journal ArticleDOI
Targeted drug delivery strategies for precision medicines
Mandana T. Manzari,Yosi Shamay,Hiroto Kiguchi,Hiroto Kiguchi,Neal Rosen,Neal Rosen,Maurizio Scaltriti,Maurizio Scaltriti,Daniel A. Heller,Daniel A. Heller +9 more
TL;DR: This Review investigates the integration of targeted drugs, in particular, kinase inhibitors, with targeted drug delivery systems and identifies opportunities for strategies to improve the therapeutic index of cancer drugs and, consequently, clinical outcomes.
References
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Journal ArticleDOI
Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong,Le Cong,F. Ann Ran,F. Ann Ran,David M. Cox,David M. Cox,Shuailiang Lin,Shuailiang Lin,Robert P. J. Barretto,Naomi Habib,Patrick D. Hsu,Patrick D. Hsu,Xuebing Wu,Wenyan Jiang,Luciano A. Marraffini,Feng Zhang +15 more
TL;DR: The type II prokaryotic CRISPR (clustered regularly interspaced short palindromic repeats)/Cas adaptive immune system has been shown to facilitate RNA-guided site-specific DNA cleavage as discussed by the authors.
Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong,F. A. Ran,David Benjamin Turitz Cox,Shuailiang Lin,Robert P. J. Barretto,Naomi Habib,Patrick D. Hsu,Xuebing Wu,Wenyan Jiang,Luciano A. Marraffini,Feng Zhang +10 more
TL;DR: Two different type II CRISPR/Cas systems are engineered and it is demonstrated that Cas9 nucleases can be directed by short RNAs to induce precise cleavage at endogenous genomic loci in human and mouse cells, demonstrating easy programmability and wide applicability of the RNA-guided nuclease technology.
Journal ArticleDOI
RNA-Guided Human Genome Engineering via Cas9
Prashant Mali,Luhan Yang,Kevin M. Esvelt,John Aach,Marc Güell,James E. DiCarlo,Julie E. Norville,George M. Church,George M. Church +8 more
TL;DR: The type II bacterial CRISPR system is engineer to function with custom guide RNA (gRNA) in human cells to establish an RNA-guided editing tool for facile, robust, and multiplexable human genome engineering.
Journal ArticleDOI
One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering.
Haoyi Wang,Hui Yang,Chikdu S. Shivalila,Meelad M. Dawlaty,Albert W. Cheng,Feng Zhang,Feng Zhang,Rudolf Jaenisch +7 more
TL;DR: The CRISPR/Cas system allows the one-step generation of animals carrying mutations in multiple genes, an approach that will greatly accelerate the in vivo study of functionally redundant genes and of epistatic gene interactions.
Journal ArticleDOI
Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity
F. Ann Ran,Patrick D. Hsu,Chie Yu Lin,Jonathan S. Gootenberg,Silvana Konermann,Alexandro E. Trevino,David A. Scott,Azusa Inoue,Shogo Matoba,Yi Zhang,Feng Zhang +10 more
TL;DR: In this paper, an approach that combines a Cas9 nickase mutant with paired guide RNAs to introduce targeted double-strand breaks is described. But the approach is limited to mouse zygotes.