标题
The promise and challenge of therapeutic genome editing
作者
关键词
-
出版物
NATURE
Volume 578, Issue 7794, Pages 229-236
出版商
Springer Science and Business Media LLC
发表日期
2020-02-13
DOI
10.1038/s41586-020-1978-5
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- A natural regulatory mutation in the proximal promoter elevates fetal globin expression by creating a de novo GATA1 site
- (2019) Gabriella E. Martyn et al. BLOOD
- The CRISPR/Cas9 system and its applications in crop genome editing
- (2019) Aili Bao et al. CRITICAL REVIEWS IN BIOTECHNOLOGY
- Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10
- (2019) Morgan L. Maeder et al. NATURE MEDICINE
- Delivery technologies for cancer immunotherapy
- (2019) Rachel S. Riley et al. NATURE REVIEWS DRUG DISCOVERY
- Standardized, Scalable, and Timely Flexible Adeno-Associated Virus Vector Production Using Frozen High-Density HEK-293 Cell Stocks and CELLdiscs
- (2019) Benjamin Strobel et al. Human Gene Therapy Methods
- A New Class of Medicines through DNA Editing
- (2019) Matthew H. Porteus NEW ENGLAND JOURNAL OF MEDICINE
- Identification of preexisting adaptive immunity to Cas9 proteins in humans
- (2019) Carsten T. Charlesworth et al. NATURE MEDICINE
- Highly efficient therapeutic gene editing of human hematopoietic stem cells
- (2019) Yuxuan Wu et al. NATURE MEDICINE
- Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response
- (2019) Giulia Schiroli et al. Cell Stem Cell
- Evaluating and Enhancing Target Specificity of Gene-Editing Nucleases and Deaminases
- (2019) Daesik Kim et al. Annual Review of Biochemistry
- Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors
- (2019) Julian Grünewald et al. NATURE
- The next generation of CRISPR–Cas technologies and applications
- (2019) Adrian Pickar-Oliver et al. NATURE REVIEWS MOLECULAR CELL BIOLOGY
- Off-target RNA mutation induced by DNA base editing and its elimination by mutagenesis
- (2019) Changyang Zhou et al. NATURE
- 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
- Sarcomere Gene Mutation correction
- (2018) Sanjiv Kaul et al. EUROPEAN HEART JOURNAL
- A CRISPR–dCas Toolbox for Genetic Engineering and Synthetic Biology
- (2018) Xiaoshu Xu et al. JOURNAL OF MOLECULAR BIOLOGY
- Receptor-Mediated Delivery of CRISPR-Cas9 Endonuclease for Cell-Type-Specific Gene Editing
- (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
- CRISPR–Cas9 genome editing induces a p53-mediated DNA damage response
- (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
- Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population
- (2018) Vijaya L. Simhadri et al. Molecular Therapy-Methods & Clinical Development
- Functional Genetic Variants Revealed by Massively Parallel Precise Genome Editing
- (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
- Inter-homologue repair in fertilized human eggs?
- (2018) Dieter Egli et al. NATURE
- Generation of a TP53-modified porcine cancer model by CRISPR/Cas9-mediated gene modification in porcine zygotes via electroporation
- (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
- Inhibition of HBV Expression in HBV Transgenic Mice Using AAV-Delivered CRISPR-SaCas9
- (2018) Hao Li et al. Frontiers in Immunology
- CRISPR Correction of Duchenne Muscular Dystrophy
- (2018) Yi-Li Min et al. Annual Review of Medicine
- Potent Protein Delivery into Mammalian Cells via a Supercharged Polypeptide
- (2018) Jun Yin et al. JOURNAL OF THE AMERICAN CHEMICAL SOCIETY
- CRISPR-Based Technologies: Impact of RNA-Targeting Systems
- (2018) Michael P. Terns MOLECULAR CELL
- High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population
- (2018) Dimitrios L. Wagner et al. NATURE MEDICINE
- Emerging Strategies in the Treatment of Duchenne Muscular Dystrophy
- (2018) Perry B. Shieh Neurotherapeutics
- High-throughput in vivo screen of functional mRNA delivery identifies nanoparticles for endothelial cell gene editing
- (2018) Cory D. Sago et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Improving Gene Editing Outcomes in Human Hematopoietic Stem and Progenitor Cells by Temporal Control of DNA Repair
- (2018) Anastasia Lomova et al. STEM CELLS
- Advances of blood cell-based drug delivery systems
- (2017) Yanan Sun et al. EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES
- CRISPR/Cas9-mediated gene editing in human zygotes using Cas9 protein
- (2017) Lichun Tang et al. MOLECULAR GENETICS AND GENOMICS
- Efficient intracellular delivery of biomacromolecules employing clusters of zinc oxide nanowires
- (2017) Prashant Sharma et al. Nanoscale
- Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents
- (2017) Xue Gao et al. NATURE
- Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage
- (2017) Nicole M. Gaudelli et al. NATURE
- Correction of a pathogenic gene mutation in human embryos
- (2017) Hong Ma et al. NATURE
- Genome editing reveals a role for OCT4 in human embryogenesis
- (2017) Norah M. E. Fogarty et al. NATURE
- Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes
- (2017) Brett T Staahl et al. NATURE BIOTECHNOLOGY
- Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
- (2017) Jerry R. Mendell et al. NEW ENGLAND JOURNAL OF MEDICINE
- Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy
- (2017) Leonela Amoasii et al. Science Translational Medicine
- Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells
- (2017) Waseem Qasim et al. Science Translational Medicine
- Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34 + Hematopoietic Stem and Progenitor Cells
- (2017) Kai-Hsin Chang et al. Molecular Therapy-Methods & Clinical Development
- Nanoparticles for CRISPR–Cas9 delivery
- (2017) Zachary Glass et al. Nature Biomedical Engineering
- Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair
- (2017) Kunwoo Lee et al. Nature Biomedical Engineering
- Non-Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co-Delivery of Cas9 mRNA and sgRNA
- (2016) Jason B. Miller et al. ANGEWANDTE CHEMIE-INTERNATIONAL EDITION
- Editing DNA Methylation in the Mammalian Genome
- (2016) X. Shawn Liu et al. CELL
- Genetic disruption of theKLF1gene to overexpress the γ-globin gene using the CRISPR/Cas9system
- (2016) Laleh Shariati et al. JOURNAL OF GENE MEDICINE
- CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice
- (2016) Li Xu et al. MOLECULAR THERAPY
- CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells
- (2016) Daniel P. Dever et al. NATURE
- Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
- (2016) Alexis C. Komor et al. NATURE
- A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice
- (2016) Yang Yang et al. NATURE BIOTECHNOLOGY
- Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles
- (2016) Ming Wang et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems
- (2016) K. Nishida et al. SCIENCE
- Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells
- (2016) M. A. DeWitt et al. Science Translational Medicine
- A Cas9 Ribonucleoprotein Platform for Functional Genetic Studies of HIV-Host Interactions in Primary Human T Cells
- (2016) Judd F. Hultquist et al. Cell Reports
- Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases
- (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
Become a Peeref-certified reviewer
The Peeref Institute provides free reviewer training that teaches the core competencies of the academic peer review process.
Get StartedAsk a Question. Answer a Question.
Quickly pose questions to the entire community. Debate answers and get clarity on the most important issues facing researchers.
Get Started