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Literature DB >> 33077937

Base editing: advances and therapeutic opportunities.

Elizabeth M Porto¹, Alexis C Komor², Ian M Slaymaker³, Gene W Yeo^4,5,6.

Abstract

Base editing - the introduction of single-nucleotide variants (SNVs) into DNA or RNA in living cells - is one of the most recent advances in the field of genome editing. As around half of known pathogenic genetic variants are due to SNVs, base editing holds great potential for the treatment of numerous genetic diseases, through either temporary RNA or permanent DNA base alterations. Recent advances in the specificity, efficiency, precision and delivery of DNA and RNA base editors are revealing exciting therapeutic opportunities for these technologies. We expect the correction of single point mutations will be a major focus of future precision medicine.

Entities: Chemical

Mesh：

Year: 2020 PMID： 33077937 PMCID： PMC7721651 DOI： 10.1038/s41573-020-0084-6

Source DB: PubMed Journal: Nat Rev Drug Discov ISSN： 1474-1776 Impact factor: 112.288

238 in total

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Authors: Feng Zhang; James R Lupski
Journal: Hum Mol Genet Date: 2015-07-07 Impact factor: 6.150

2. Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria.

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Journal: Proc Natl Acad Sci U S A Date: 2012-09-04 Impact factor: 11.205

3. Genomic characterization of metastatic breast cancers.

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Journal: Nature Date: 2019-05-22 Impact factor: 49.962

4. Efficient and allele-specific genome editing of disease loci in human iPSCs.

Authors: Cory Smith; Leire Abalde-Atristain; Chaoxia He; Brett R Brodsky; Evan M Braunstein; Pooja Chaudhari; Yoon-Young Jang; Linzhao Cheng; Zhaohui Ye
Journal: Mol Ther Date: 2014-11-24 Impact factor: 11.454

5. 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

6. RNA-guided human genome engineering via Cas9.

Authors: Prashant Mali; Luhan Yang; Kevin M Esvelt; John Aach; Marc Guell; James E DiCarlo; Julie E Norville; George M Church
Journal: Science Date: 2013-01-03 Impact factor: 47.728

Review 7. CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes.

Authors: Alexis C Komor; Ahmed H Badran; David R Liu
Journal: Cell Date: 2016-11-17 Impact factor: 41.582

8. A general framework for estimating the relative pathogenicity of human genetic variants.

Authors: Martin Kircher; Daniela M Witten; Preti Jain; Brian J O'Roak; Gregory M Cooper; Jay Shendure
Journal: Nat Genet Date: 2014-02-02 Impact factor: 38.330

Review 9. Single nucleotide variations: biological impact and theoretical interpretation.

Authors: Panagiotis Katsonis; Amanda Koire; Stephen Joseph Wilson; Teng-Kuei Hsu; Rhonald C Lua; Angela Dawn Wilkins; Olivier Lichtarge
Journal: Protein Sci Date: 2014-10-20 Impact factor: 6.725

10. A global reference for human genetic variation.

Authors: Adam Auton; Lisa D Brooks; Richard M Durbin; Erik P Garrison; Hyun Min Kang; Jan O Korbel; Jonathan L Marchini; Shane McCarthy; Gil A McVean; Gonçalo R Abecasis
Journal: Nature Date: 2015-10-01 Impact factor: 49.962

38 in total

1. Adenine base-editing-mediated exon skipping induces gene knockout in cultured pig cells.

Authors: Xiang-Xing Zhu; Jia-Sheng Pan; Tao Lin; Ye-Cheng Yang; Qiu-Yan Huang; Shuai-Peng Yang; Zi-Xiao Qu; Zi-Sheng Lin; Jian-Cong Wen; Ai-Fen Yan; Juan Feng; Lian Liu; Xiao-Li Zhang; Jia-Hong Lu; Dong-Sheng Tang
Journal: Biotechnol Lett Date: 2022-01-08 Impact factor: 2.461

2. Targeting Alternative Splicing for Therapeutic Interventions.

Authors: Jessica L Centa; Michelle L Hastings
Journal: Methods Mol Biol Date: 2022

3. Hypercompact adenine base editors based on transposase B guided by engineered RNA.

Authors: Do Yon Kim; Yuhee Chung; Yujin Lee; Dongmin Jeong; Kwang-Hyun Park; Hyun Jung Chin; Jeong Mi Lee; Seyeon Park; Sumin Ko; Jeong-Heon Ko; Yong-Sam Kim
Journal: Nat Chem Biol Date: 2022-08-01 Impact factor: 16.174

4. High-fidelity Cas13 variants for targeted RNA degradation with minimal collateral effects.

Authors: Huawei Tong; Jia Huang; Qingquan Xiao; Bingbing He; Xue Dong; Yuanhua Liu; Xiali Yang; Dingyi Han; Zikang Wang; Xuchen Wang; Wenqin Ying; Runze Zhang; Yu Wei; Chunlong Xu; Yingsi Zhou; Yanfei Li; Minqing Cai; Qifang Wang; Mingxing Xue; Guoling Li; Kailun Fang; Hainan Zhang; Hui Yang
Journal: Nat Biotechnol Date: 2022-08-11 Impact factor: 68.164

Review 5. Developing Bottom-Up Induced Pluripotent Stem Cell Derived Solid Tumor Models Using Precision Genome Editing Technologies.

Authors: Kelsie L Becklin; Garrett M Draper; Rebecca A Madden; Mitchell G Kluesner; Tomoyuki Koga; Miller Huang; William A Weiss; Logan G Spector; David A Largaespada; Branden S Moriarity; Beau R Webber
Journal: CRISPR J Date: 2022-08