Literature DB >> 28191903

Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes.

Brett T Staahl1, Madhurima Benekareddy2, Claire Coulon-Bainier2, Ashwin A Banfal1, Stephen N Floor1, Jennifer K Sabo1, Cole Urnes1, Gabriela Acevedo Munares1, Anirvan Ghosh2,3, Jennifer A Doudna1,4,5,6,7.   

Abstract

We demonstrate editing of post-mitotic neurons in the adult mouse brain following injection of Cas9 ribonucleoprotein (RNP) complexes in the hippocampus, striatum and cortex. Engineered variants of Cas9 with multiple SV40 nuclear localization sequences enabled a tenfold increase in the efficiency of neuronal editing in vivo. These advances indicate the potential of genome editing in the brain to correct or inactivate the underlying genetic causes of neurological diseases.

Entities:  

Mesh：

Substances：

Year:  2017        PMID： 28191903      PMCID： PMC6649674          DOI： 10.1038/nbt.3806

Source DB:  PubMed          Journal:  Nat Biotechnol        ISSN： 1087-0156            Impact factor:   54.908


  29 in total

1.  Optimization of nonviral gene transfer of vascular smooth muscle cells in vitro and in vivo.

Authors:  S Armeanu; J Pelisek; E Krausz; A Fuchs; D Groth; R Curth; O Keil; J Quilici; P H Rolland; R Reszka; S Nikol
Journal:  Mol Ther       Date:  2000-04       Impact factor: 11.454

2.  Engineering and characterization of a superfolder green fluorescent protein.

Authors:  Jean-Denis Pédelacq; Stéphanie Cabantous; Timothy Tran; Thomas C Terwilliger; Geoffrey S Waldo
Journal:  Nat Biotechnol       Date:  2005-12-20       Impact factor: 54.908

3.  Oxygen radical-mediated pulmonary toxicity induced by some cationic liposomes.

Authors:  S Dokka; D Toledo; X Shi; V Castranova; Y Rojanasakul
Journal:  Pharm Res       Date:  2000-05       Impact factor: 4.200

4.  A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity.

Authors:  Martin Jinek; Krzysztof Chylinski; Ines Fonfara; Michael Hauer; Jennifer A Doudna; Emmanuelle Charpentier
Journal:  Science       Date:  2012-06-28       Impact factor: 47.728

5.  A robust and high-throughput Cre reporting and characterization system for the whole mouse brain.

Authors:  Linda Madisen; Theresa A Zwingman; Susan M Sunkin; Seung Wook Oh; Hatim A Zariwala; Hong Gu; Lydia L Ng; Richard D Palmiter; Michael J Hawrylycz; Allan R Jones; Ed S Lein; Hongkui Zeng
Journal:  Nat Neurosci       Date:  2009-12-20       Impact factor: 24.884

6.  Multiplex genome engineering using CRISPR/Cas systems.

Authors:  Le Cong; F Ann Ran; David Cox; Shuailiang Lin; Robert Barretto; Naomi Habib; Patrick D Hsu; Xuebing Wu; Wenyan Jiang; Luciano A Marraffini; Feng Zhang
Journal:  Science       Date:  2013-01-03       Impact factor: 47.728

7.  DNA targeting specificity of RNA-guided Cas9 nucleases.

Authors:  Patrick D Hsu; David A Scott; Joshua A Weinstein; F Ann Ran; Silvana Konermann; Vineeta Agarwala; Yinqing Li; Eli J Fine; Xuebing Wu; Ophir Shalem; Thomas J Cradick; Luciano A Marraffini; Gang Bao; Feng Zhang
Journal:  Nat Biotechnol       Date:  2013-07-21       Impact factor: 54.908

8.  CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes.

Authors:  Luke A Gilbert; Matthew H Larson; Leonardo Morsut; Zairan Liu; Gloria A Brar; Sandra E Torres; Noam Stern-Ginossar; Onn Brandman; Evan H Whitehead; Jennifer A Doudna; Wendell A Lim; Jonathan S Weissman; Lei S Qi
Journal:  Cell       Date:  2013-07-11       Impact factor: 41.582

9.  CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering.

Authors:  Prashant Mali; John Aach; P Benjamin Stranges; Kevin M Esvelt; Mark Moosburner; Sriram Kosuri; Luhan Yang; George M Church
Journal:  Nat Biotechnol       Date:  2013-08-01       Impact factor: 54.908

10.  RNA-programmed genome editing in human cells.

Authors:  Martin Jinek; Alexandra East; Aaron Cheng; Steven Lin; Enbo Ma; Jennifer Doudna
Journal:  Elife       Date:  2013-01-29       Impact factor: 8.140
View more
  108 in total

1.  Fluorescent labeling of CRISPR/Cas9 RNP for gene knockout in HSPCs and iPSCs reveals an essential role for GADD45b in stress response.

Authors:  Masoud Nasri; Perihan Mir; Benjamin Dannenmann; Diana Amend; Tessa Skroblyn; Yun Xu; Klaus Schulze-Osthoff; Maksim Klimiankou; Karl Welte; Julia Skokowa
Journal:  Blood Adv       Date:  2019-01-08

Review 2.  Gene-edited CRISPy Critters for alcohol research.

Authors:  Gregg E Homanics
Journal:  Alcohol       Date:  2018-03-07       Impact factor: 2.405

3.  Chemically Modified Cpf1-CRISPR RNAs Mediate Efficient Genome Editing in Mammalian Cells.

Authors:  Moira A McMahon; Thazha P Prakash; Don W Cleveland; C Frank Bennett; Meghdad Rahdar
Journal:  Mol Ther       Date:  2018-03-06       Impact factor: 11.454

4.  Spatiotemporal Control of CRISPR/Cas9 Function in Cells and Zebrafish using Light-Activated Guide RNA.

Authors:  Wenyuan Zhou; Wes Brown; Anirban Bardhan; Michael Delaney; Amber S Ilk; Randy R Rauen; Shoeb I Kahn; Michael Tsang; Alexander Deiters
Journal:  Angew Chem Int Ed Engl       Date:  2020-04-06       Impact factor: 15.336

Review 5.  Cas9 Ribonucleoprotein Complex Delivery: Methods and Applications for Neuroinflammation.

Authors:  Lee A Campbell; Christopher T Richie; Nishad S Maggirwar; Brandon K Harvey
Journal:  J Neuroimmune Pharmacol       Date:  2019-06-06       Impact factor: 4.147

6.  A peptide delivery system sneaks CRISPR into cells.

Authors:  Xingang Guan; Zhimin Luo; Wujin Sun
Journal:  J Biol Chem       Date:  2018-11-02       Impact factor: 5.157

7.  Intracellular Delivery of His-Tagged Genome-Editing Proteins Enabled by Nitrilotriacetic Acid-Containing Lipidoid Nanoparticles.

Authors:  Yamin Li; Alice Chukun Li; Qiaobing Xu
Journal:  Adv Healthc Mater       Date:  2018-11-22       Impact factor: 9.933

8.  Targeted deletion of an NRL- and CRX-regulated alternative promoter specifically silences FERM and PDZ domain containing 1 (Frmpd1) in rod photoreceptors.

Authors:  Christie K Campla; Hannah Mast; Lijin Dong; Jingqi Lei; Stephanie Halford; Sumathi Sekaran; Anand Swaroop
Journal:  Hum Mol Genet       Date:  2019-03-01       Impact factor: 6.150

Review 9.  Molecular tools to elucidate factors regulating alcohol use.

Authors:  Marian L Logrip
Journal:  Alcohol       Date:  2018-03-20       Impact factor: 2.405

10.  Nanoparticles for CRISPR-Cas9 delivery.

Authors:  Zachary Glass; Yamin Li; Qiaobing Xu
Journal:  Nat Biomed Eng       Date:  2017-11-10       Impact factor: 25.671
View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.