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Title
The promise and challenge of therapeutic genome editing
Authors
Keywords
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Journal
NATURE
Volume 578, Issue 7794, Pages 229-236
Publisher
Springer Science and Business Media LLC
Online
2020-02-13
DOI
10.1038/s41586-020-1978-5
References
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Note: Only part of the references are listed.- A natural regulatory mutation in the proximal promoter elevates fetal globin expression by creating a de novo GATA1 site
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- (2019) Carsten T. Charlesworth et al. NATURE MEDICINE
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- Gene Therapy
- (2019) Katherine A. High et al. NEW ENGLAND JOURNAL OF MEDICINE
- CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia
- (2019) Lei Xu et al. NEW ENGLAND JOURNAL OF MEDICINE
- Search-and-replace genome editing without double-strand breaks or donor DNA
- (2019) Andrew V. Anzalone et al. NATURE
- Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus
- (2018) Chiara Antoniani et al. BLOOD
- Direct Promoter Repression by BCL11A Controls the Fetal to Adult Hemoglobin Switch
- (2018) Nan Liu et al. CELL
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- (2018) Sanjiv Kaul et al. EUROPEAN HEART JOURNAL
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- (2018) Romain Rouet et al. JOURNAL OF THE AMERICAN CHEMICAL SOCIETY
- Cas13d Is a Compact RNA-Targeting Type VI CRISPR Effector Positively Modulated by a WYL-Domain-Containing Accessory Protein
- (2018) Winston X. Yan et al. MOLECULAR CELL
- Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing
- (2018) Kanut Laoharawee et al. MOLECULAR THERAPY
- Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements
- (2018) Michael Kosicki et al. NATURE BIOTECHNOLOGY
- Natural regulatory mutations elevate the fetal globin gene via disruption of BCL11A or ZBTB7A binding
- (2018) Gabriella E. Martyn et al. NATURE GENETICS
- p53 inhibits CRISPR–Cas9 engineering in human pluripotent stem cells
- (2018) Robert J. Ihry et al. NATURE MEDICINE
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- (2018) Emma Haapaniemi et al. NATURE MEDICINE
- Domain-focused CRISPR screen identifies HRI as a fetal hemoglobin regulator in human erythroid cells
- (2018) Jeremy D. Grevet et al. SCIENCE
- A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing
- (2018) Jonathan D. Finn et al. Cell Reports
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- (2018) Vijaya L. Simhadri et al. Molecular Therapy-Methods & Clinical Development
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- (2018) Eilon Sharon et al. CELL
- Characterization and Repurposing of Type I and Type II CRISPR-Cas Systems in Bacteria
- (2018) Claudio Hidalgo-Cantabrana et al. JOURNAL OF MOLECULAR BIOLOGY
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- (2018) Dieter Egli et al. NATURE
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- (2018) Fuminori Tanihara et al. PLoS One
- Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
- (2018) Leonela Amoasii et al. SCIENCE
- CRISPR-Cas guides the future of genetic engineering
- (2018) Gavin J. Knott et al. SCIENCE
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- (2018) Hao Li et al. Frontiers in Immunology
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- (2018) Yi-Li Min et al. Annual Review of Medicine
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- (2017) Lichun Tang et al. MOLECULAR GENETICS AND GENOMICS
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- (2016) Alexis C. Komor et al. NATURE
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- Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems
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- Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells
- (2016) M. A. DeWitt et al. Science Translational Medicine
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- (2016) Judd F. Hultquist et al. Cell Reports
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- (2016) Carmen F Bjurström et al. Molecular Therapy-Nucleic Acids
- In vivo genome editing of the albumin locus as a platform for protein replacement therapy
- (2015) R. Sharma et al. BLOOD
- MLL leukemia induction by genome editing of human CD34+ hematopoietic cells
- (2015) C. Buechele et al. BLOOD
- Multiplex Genome-Edited T-cell Manufacturing Platform for “Off-the-Shelf” Adoptive T-cell Immunotherapies
- (2015) Laurent Poirot et al. CANCER RESEARCH
- Generation of knock-in primary human T cells using Cas9 ribonucleoproteins
- (2015) Kathrin Schumann et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- In vivo gene editing in dystrophic mouse muscle and muscle stem cells
- (2015) M. Tabebordbar et al. SCIENCE
- In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
- (2015) C. E. Nelson et al. SCIENCE
- CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes
- (2015) Puping Liang et al. Protein & Cell
- Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation
- (2014) Luke A. Gilbert et al. CELL
- Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins
- (2014) S. Kim et al. GENOME RESEARCH
- Gold Nanoparticles for Nucleic Acid Delivery
- (2014) Ya Ding et al. MOLECULAR THERAPY
- In vivo engineering of oncogenic chromosomal rearrangements with the CRISPR/Cas9 system
- (2014) Danilo Maddalo et al. NATURE
- Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo
- (2014) John A Zuris et al. NATURE BIOTECHNOLOGY
- Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA
- (2014) C. Long et al. SCIENCE
- Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression
- (2013) Lei S. Qi et al. CELL
- CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes
- (2013) Luke A. Gilbert et al. CELL
- Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease
- (2013) Seung Woo Cho et al. NATURE BIOTECHNOLOGY
- RNA-Guided Human Genome Engineering via Cas9
- (2013) P. Mali et al. SCIENCE
- Multiplex Genome Engineering Using CRISPR/Cas Systems
- (2013) L. Cong et al. SCIENCE
- RNA-programmed genome editing in human cells
- (2013) Martin Jinek et al. eLife
- Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria
- (2012) G. Gasiunas et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
- (2012) M. Jinek et al. SCIENCE
- A TALE nuclease architecture for efficient genome editing
- (2010) Jeffrey C Miller et al. NATURE BIOTECHNOLOGY
- Mouse Embryonic Stem Cells, but Not Somatic Cells, Predominantly Use Homologous Recombination to Repair Double-Strand DNA Breaks
- (2010) Elisia D. Tichy et al. STEM CELLS AND DEVELOPMENT
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