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Literature DB >> 28931646

Genome editing technologies and their potential to treat neurologic disease.

Nicolas N Madigan¹, Nathan P Staff², Anthony J Windebank², Eduardo E Benarroch².

Abstract

Entities: Disease

Mesh：

Year: 2017 PMID： 28931646 PMCID： PMC5644461 DOI： 10.1212/WNL.0000000000004558

Source DB: PubMed Journal: Neurology ISSN： 0028-3878 Impact factor: 9.910

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56 in total

1. Correction of a pathogenic gene mutation in human embryos.

Authors: Hong Ma; Nuria Marti-Gutierrez; Sang-Wook Park; Jun Wu; Yeonmi Lee; Keiichiro Suzuki; Amy Koski; Dongmei Ji; Tomonari Hayama; Riffat Ahmed; Hayley Darby; Crystal Van Dyken; Ying Li; Eunju Kang; A-Reum Park; Daesik Kim; Sang-Tae Kim; Jianhui Gong; Ying Gu; Xun Xu; David Battaglia; Sacha A Krieg; David M Lee; Diana H Wu; Don P Wolf; Stephen B Heitner; Juan Carlos Izpisua Belmonte; Paula Amato; Jin-Soo Kim; Sanjiv Kaul; Shoukhrat Mitalipov
Journal: Nature Date: 2017-08-02 Impact factor: 49.962

2. A yellow light for embryo editing.

Authors: Jocelyn Kaiser
Journal: Science Date: 2017-02-17 Impact factor: 47.728

3. In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.

Authors: Christopher E Nelson; Chady H Hakim; David G Ousterout; Pratiksha I Thakore; Eirik A Moreb; Ruth M Castellanos Rivera; Sarina Madhavan; Xiufang Pan; F Ann Ran; Winston X Yan; Aravind Asokan; Feng Zhang; Dongsheng Duan; Charles A Gersbach
Journal: Science Date: 2015-12-31 Impact factor: 47.728

4. cGMP-Compliant Expansion of Human iPSC Cultures as Adherent Monolayers.

Authors: Ann M Parr; Patrick J Walsh; Vincent Truong; James R Dutton
Journal: Methods Mol Biol Date: 2016

5. Small molecules enhance CRISPR genome editing in pluripotent stem cells.

Authors: Chen Yu; Yanxia Liu; Tianhua Ma; Kai Liu; Shaohua Xu; Yu Zhang; Honglei Liu; Marie La Russa; Min Xie; Sheng Ding; Lei S Qi
Journal: Cell Stem Cell Date: 2015-02-05 Impact factor: 24.633

6. Discovery and Functional Characterization of Diverse Class 2 CRISPR-Cas Systems.

Authors: Sergey Shmakov; Omar O Abudayyeh; Kira S Makarova; Yuri I Wolf; Jonathan S Gootenberg; Ekaterina Semenova; Leonid Minakhin; Julia Joung; Silvana Konermann; Konstantin Severinov; Feng Zhang; Eugene V Koonin
Journal: Mol Cell Date: 2015-10-22 Impact factor: 17.970

7. Safety and tolerability of putaminal AADC gene therapy for Parkinson disease.

Authors: C W Christine; P A Starr; P S Larson; J L Eberling; W J Jagust; R A Hawkins; H F VanBrocklin; J F Wright; K S Bankiewicz; M J Aminoff
Journal: Neurology Date: 2009-10-14 Impact factor: 9.910

8. Combined small-molecule inhibition accelerates developmental timing and converts human pluripotent stem cells into nociceptors.

Authors: Stuart M Chambers; Yuchen Qi; Yvonne Mica; Gabsang Lee; Xin-Jun Zhang; Lei Niu; James Bilsland; Lishuang Cao; Edward Stevens; Paul Whiting; Song-Hai Shi; Lorenz Studer
Journal: Nat Biotechnol Date: 2012-07-01 Impact factor: 54.908

Genome editing technologies and their potential to treat neurologic disease.

1. Correction of a pathogenic gene mutation in human embryos.

2. A yellow light for embryo editing.

3. In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.

4. cGMP-Compliant Expansion of Human iPSC Cultures as Adherent Monolayers.

5. Small molecules enhance CRISPR genome editing in pluripotent stem cells.

6. Discovery and Functional Characterization of Diverse Class 2 CRISPR-Cas Systems.

7. Safety and tolerability of putaminal AADC gene therapy for Parkinson disease.

8. Combined small-molecule inhibition accelerates developmental timing and converts human pluripotent stem cells into nociceptors.

9. Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery.

10. Rapid characterization of CRISPR-Cas9 protospacer adjacent motif sequence elements.

1. Protein and mRNA Delivery Enabled by Cholesteryl-Based Biodegradable Lipidoid Nanoparticles.

2. Recent Scientific Breakthroughs Applying CRISPR Gene Editing in Neurological Disorders.