标题
CRISPR/Cas9-mediated correction of human genetic disease
作者
关键词
CRISPR/Cas9, genome editing, genetic disease, gene therapy
出版物
Science China-Life Sciences
Volume 60, Issue 5, Pages 447-457
出版商
Springer Nature
发表日期
2017-05-22
DOI
10.1007/s11427-017-9032-4
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。- CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice
- (2016) Li Xu et al. MOLECULAR THERAPY
- A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice
- (2016) Yang Yang et al. NATURE BIOTECHNOLOGY
- Pluripotent stem cells in disease modelling and drug discovery
- (2016) Yishai Avior et al. NATURE REVIEWS MOLECULAR CELL BIOLOGY
- Pluripotent stem cells progressing to the clinic
- (2016) Alan Trounson et al. NATURE REVIEWS MOLECULAR CELL BIOLOGY
- A novel RNA-guided RNA-targeting CRISPR tool
- (2016) Dandan Zhang et al. Science China-Life Sciences
- DNA-guided genome editing tool
- (2016) Dandan Zhang et al. Science China-Life Sciences
- From the first human gene-editing to the birth of three-parent baby
- (2016) Xiaoxue Zhang et al. Science China-Life Sciences
- Advances in therapeutic CRISPR/Cas9 genome editing
- (2016) Nataša Savić et al. Translational Research
- Distribution of AAV8 particles in cell lysates and culture media changes with time and is dependent on the recombinant vector
- (2016) Bryan A Piras et al. Molecular Therapy-Methods & Clinical Development
- CRISPR/Cas9-mediated gene knockout in the mouse brain using in utero electroporation
- (2016) Yohei Shinmyo et al. Scientific Reports
- Precision Medicine: Genetic Repair of Retinitis Pigmentosa in Patient-Derived Stem Cells
- (2016) Alexander G. Bassuk et al. Scientific Reports
- Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9
- (2015) Chul-Yong Park et al. Cell Stem Cell
- CRISPR-mediated genotypic and phenotypic correction of a chronic granulomatous disease mutation in human iPS cells
- (2015) Rowan Flynn et al. EXPERIMENTAL HEMATOLOGY
- Genome Editing of the Germline: Broadening the Discussion
- (2015) Matthew H Porteus et al. MOLECULAR THERAPY
- In vivo genome editing using Staphylococcus aureus Cas9
- (2015) F. Ann Ran et al. NATURE
- Current status of pluripotent stem cells: moving the first therapies to the clinic
- (2015) Erin A. Kimbrel et al. NATURE REVIEWS DRUG DISCOVERY
- Model animals and their applications
- (2015) Xiang Gao Science China-Life Sciences
- Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation
- (2015) Xiaosong Huang et al. STEM CELLS
- Improved Hematopoietic Differentiation Efficiency of Gene-Corrected Beta-Thalassemia Induced Pluripotent Stem Cells by CRISPR/Cas9 System
- (2015) Bing Song et al. STEM CELLS AND DEVELOPMENT
- Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy
- (2015) David G. Ousterout et al. Nature Communications
- Modeling Human Severe Combined Immunodeficiency and Correction by CRISPR/Cas9-Enhanced Gene Targeting
- (2015) Chia-Wei Chang et al. Cell Reports
- Naïve Induced Pluripotent Stem Cells Generated From β-Thalassemia Fibroblasts Allow Efficient Gene Correction With CRISPR/Cas9
- (2015) Yuanyuan Yang et al. Stem Cells Translational Medicine
- Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9
- (2015) Hongmei Lisa Li et al. Stem Cell Reports
- CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling
- (2014) Randall J. Platt et al. CELL
- Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing
- (2014) Qiurong Ding et al. CIRCULATION RESEARCH
- Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 andpiggyBac
- (2014) Fei Xie et al. GENOME RESEARCH
- In vivo engineering of oncogenic chromosomal rearrangements with the CRISPR/Cas9 system
- (2014) Danilo Maddalo et al. NATURE
- In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9
- (2014) Lukasz Swiech et al. NATURE BIOTECHNOLOGY
- Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
- (2014) Hao Yin et al. NATURE BIOTECHNOLOGY
- Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA
- (2014) C. Long et al. SCIENCE
- Cell therapy for macular degeneration—first phase I/II pluripotent stem cell-based clinical trial shows promise
- (2014) ZhiGuo Chen et al. Science China-Life Sciences
- Acquired Resistance of EGFR-Mutant Lung Adenocarcinomas to Afatinib plus Cetuximab Is Associated with Activation of mTORC1
- (2014) Valentina Pirazzoli et al. Cell Reports
- Simple and Rapid In Vivo Generation of Chromosomal Rearrangements using CRISPR/Cas9 Technology
- (2014) Rafael B. Blasco et al. Cell Reports
- One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering
- (2013) Haoyi Wang et al. CELL
- One-Step Generation of Mice Carrying Reporter and Conditional Alleles by CRISPR/Cas-Mediated Genome Engineering
- (2013) Hui Yang et al. CELL
- Genome editing with RNA-guided Cas9 nuclease in Zebrafish embryos
- (2013) Nannan Chang et al. CELL RESEARCH
- Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients
- (2013) Gerald Schwank et al. Cell Stem Cell
- Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9
- (2013) Yuxuan Wu et al. Cell Stem Cell
- Highly Efficient Genome Modifications Mediated by CRISPR/Cas9 in Drosophila
- (2013) Z. Yu et al. GENETICS
- Selection and evaluation of clinically relevant AAV variants in a xenograft liver model
- (2013) Leszek Lisowski et al. NATURE
- Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease
- (2013) Seung Woo Cho et al. NATURE BIOTECHNOLOGY
- Simultaneous generation and germline transmission of multiple gene mutations in rat using CRISPR-Cas systems
- (2013) Wei Li et al. NATURE BIOTECHNOLOGY
- Efficient genome editing in zebrafish using a CRISPR-Cas system
- (2013) Woong Y Hwang et al. NATURE BIOTECHNOLOGY
- Engineering the Caenorhabditis elegans genome using Cas9-triggered homologous recombination
- (2013) Daniel J Dickinson et al. NATURE METHODS
- Cas9 as a versatile tool for engineering biology
- (2013) Prashant Mali et al. NATURE METHODS
- Efficient multiplex biallelic zebrafish genome editing using a CRISPR nuclease system
- (2013) Li-En Jao et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- 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
- An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells
- (2013) Antonio Filareto et al. Nature Communications
- RNA-programmed genome editing in human cells
- (2013) Martin Jinek et al. eLife
- The AAV Vector Toolkit: Poised at the Clinical Crossroads
- (2012) Aravind Asokan et al. MOLECULAR THERAPY
- The promise of induced pluripotent stem cells in research and therapy
- (2012) Daisy A. Robinton et al. NATURE
- De novo mutations in human genetic disease
- (2012) Joris A. Veltman et al. NATURE REVIEWS GENETICS
- Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery
- (2012) Y. Aoki et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- iPSCs: Unstable Origins?
- (2011) Sayandip Mukherjee et al. MOLECULAR THERAPY
- Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD
- (2011) Christophe Pichavant et al. MOLECULAR THERAPY
- High-frequency genome editing using ssDNA oligonucleotides with zinc-finger nucleases
- (2011) Fuqiang Chen et al. NATURE METHODS
- Family-based designs for genome-wide association studies
- (2011) Jurg Ott et al. NATURE REVIEWS GENETICS
- Exome sequencing as a tool for Mendelian disease gene discovery
- (2011) Michael J. Bamshad et al. NATURE REVIEWS GENETICS
- Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
- (2011) Federico Mingozzi et al. NATURE REVIEWS GENETICS
- Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
- (2011) Amit C. Nathwani et al. NEW ENGLAND JOURNAL OF MEDICINE
- Unlocking Mendelian disease using exome sequencing
- (2011) Christian Gilissen et al. GENOME BIOLOGY
- Targeted Genome Modification in Mice Using Zinc-Finger Nucleases
- (2010) I. D. Carbery et al. GENETICS
- The Status of Exon Skipping as a Therapeutic Approach to Duchenne Muscular Dystrophy
- (2010) Qi-Long Lu et al. MOLECULAR THERAPY
- DNA resection in eukaryotes: deciding how to fix the break
- (2010) Pablo Huertas NATURE STRUCTURAL & MOLECULAR BIOLOGY
- Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo
- (2009) Nicole K. Paulk et al. HEPATOLOGY
- Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations
- (2009) Annemieke Aartsma-Rus et al. HUMAN MUTATION
- Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells
- (2009) Ángel Raya et al. NATURE
- Human genetic variation and its contribution to complex traits
- (2009) Kelly A. Frazer et al. NATURE REVIEWS GENETICS
- Recombinational DNA repair in a cellular context: a search for the homology search
- (2009) Allon Weiner et al. NATURE REVIEWS MICROBIOLOGY
- Disease-Specific Induced Pluripotent Stem Cells
- (2008) In-Hyun Park et al. CELL
- Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection
- (2008) Carmela Zincarelli et al. MOLECULAR THERAPY
Find Funding. Review Successful Grants.
Explore over 25,000 new funding opportunities and over 6,000,000 successful grants.
ExploreBecome a Peeref-certified reviewer
The Peeref Institute provides free reviewer training that teaches the core competencies of the academic peer review process.
Get Started