Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells
出版年份 2016 全文链接
标题
Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells
作者
关键词
-
出版物
Science Translational Medicine
Volume 8, Issue 360, Pages 360ra134-360ra134
出版商
American Association for the Advancement of Science (AAAS)
发表日期
2016-10-13
DOI
10.1126/scitranslmed.aaf9336
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。- Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease
- (2016) Mark C. Walters et al. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
- High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects
- (2016) Benjamin P. Kleinstiver et al. NATURE
- Enhancing homology-directed genome editing by catalytically active and inactive CRISPR-Cas9 using asymmetric donor DNA
- (2016) Christopher D Richardson et al. NATURE BIOTECHNOLOGY
- Near-optimal probabilistic RNA-seq quantification
- (2016) Nicolas L Bray et al. NATURE BIOTECHNOLOGY
- Non-homologous DNA increases gene disruption efficiency by altering DNA repair outcomes
- (2016) C. D. Richardson et al. Nature Communications
- Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells
- (2015) M. D. Hoban et al. BLOOD
- Rapid and highly efficient mammalian cell engineering via Cas9 protein transfection
- (2015) Xiquan Liang et al. JOURNAL OF BIOTECHNOLOGY
- Genome Editing Technologies: Defining a Path to Clinic
- (2015) Jacqueline Corrigan-Curay et al. MOLECULAR THERAPY
- BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis
- (2015) Matthew C. Canver et al. NATURE
- Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining
- (2015) Takeshi Maruyama et al. NATURE BIOTECHNOLOGY
- Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors
- (2015) Jianbin Wang et al. NATURE BIOTECHNOLOGY
- Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells
- (2015) Ayal Hendel et al. NATURE BIOTECHNOLOGY
- Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells
- (2015) Van Trung Chu et al. NATURE BIOTECHNOLOGY
- 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
- Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template
- (2015) Blythe D. Sather et al. Science Translational Medicine
- 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
- TALEN-Mediated Generation and Genetic Correction of Disease-Specific Human Induced Pluripotent Stem Cells
- (2014) Sivaprakash Ramalingam et al. CURRENT GENE THERAPY
- Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins
- (2014) S. Kim et al. GENOME RESEARCH
- Nonmyeloablative HLA-Matched Sibling Allogeneic Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Phenotype
- (2014) Matthew M. Hsieh et al. JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
- Management of Sickle Cell Disease
- (2014) Barbara P. Yawn et al. JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
- Targeted genome editing in human repopulating haematopoietic stem cells
- (2014) Pietro Genovese et al. NATURE
- Improving CRISPR-Cas nuclease specificity using truncated guide RNAs
- (2014) Yanfang Fu et al. NATURE BIOTECHNOLOGY
- Rational design of highly active sgRNAs for CRISPR-Cas9–mediated gene inactivation
- (2014) John G Doench et al. NATURE BIOTECHNOLOGY
- A guide to genome engineering with programmable nucleases
- (2014) Hyongbum Kim et al. NATURE REVIEWS GENETICS
- The new frontier of genome engineering with CRISPR-Cas9
- (2014) J. A. Doudna et al. SCIENCE
- Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery
- (2014) Steven Lin et al. eLife
- Seamless correction of the sickle cell disease mutation of theHBBgene in human induced pluripotent stem cells using TALENs
- (2013) Ning Sun et al. BIOTECHNOLOGY AND BIOENGINEERING
- DNA targeting specificity of RNA-guided Cas9 nucleases
- (2013) Patrick D Hsu et al. NATURE BIOTECHNOLOGY
- High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells
- (2013) Yanfang Fu et al. NATURE BIOTECHNOLOGY
- CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity
- (2013) Thomas J. Cradick et al. NUCLEIC ACIDS RESEARCH
- RNA-programmed genome editing in human cells
- (2013) Martin Jinek et al. eLife
- HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease
- (2012) J. Bolanos-Meade et al. BLOOD
- Engineering humanized mice for improved hematopoietic reconstitution
- (2012) Adam C Drake et al. Cellular & Molecular Immunology
- Health-related quality of life in sickle cell disease: Past, present, and future
- (2012) Julie A. Panepinto et al. PEDIATRIC BLOOD & CANCER
- A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
- (2012) M. Jinek et al. SCIENCE
- RSEM: accurate transcript quantification from RNA-Seq data with or without a reference genome
- (2011) Bo Li et al. BMC BIOINFORMATICS
- In vivo genome editing restores haemostasis in a mouse model of haemophilia
- (2011) Hojun Li et al. NATURE
- Isolation of Single Human Hematopoietic Stem Cells Capable of Long-Term Multilineage Engraftment
- (2011) F. Notta et al. SCIENCE
Create your own webinar
Interested in hosting your own webinar? Check the schedule and propose your idea to the Peeref Content Team.
Create NowBecome a Peeref-certified reviewer
The Peeref Institute provides free reviewer training that teaches the core competencies of the academic peer review process.
Get Started