Targeted homology-directed repair in blood stem and progenitor cells with CRISPR nanoformulations
出版年份 2019 全文链接
标题
Targeted homology-directed repair in blood stem and progenitor cells with CRISPR nanoformulations
作者
关键词
-
出版物
NATURE MATERIALS
Volume -, Issue -, Pages -
出版商
Springer Science and Business Media LLC
发表日期
2019-05-28
DOI
10.1038/s41563-019-0385-5
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。- Delivering CRISPR: a review of the challenges and approaches
- (2018) Christopher A. Lino et al. DRUG DELIVERY
- Chemically Modified Cpf1-CRISPR RNAs Mediate Efficient Genome Editing in Mammalian Cells
- (2018) Moira A. McMahon et al. MOLECULAR THERAPY
- Gene therapy comes of age
- (2018) Cynthia E. Dunbar et al. SCIENCE
- Engineering the Delivery System for CRISPR-Based Genome Editing
- (2018) Zachary Glass et al. TRENDS IN BIOTECHNOLOGY
- CtIP fusion to Cas9 enhances transgene integration by homology-dependent repair
- (2018) M. Charpentier et al. Nature Communications
- A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing
- (2018) Jonathan D. Finn et al. Cell Reports
- Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours
- (2018) Bumwhee Lee et al. Nature Biomedical Engineering
- Strategies for controlling CRISPR/Cas9 off-target effects and biological variations in mammalian genome editing experiments
- (2018) Michelle L. Kimberland et al. JOURNAL OF BIOTECHNOLOGY
- Base editing: precision chemistry on the genome and transcriptome of living cells
- (2018) Holly A. Rees et al. NATURE REVIEWS GENETICS
- Improving Gene Therapy Efficiency through the Enrichment of Human Hematopoietic Stem Cells
- (2017) Katelyn E. Masiuk et al. MOLECULAR THERAPY
- CRISPR/Cas9-Mediated CCR5 Ablation in Human Hematopoietic Stem/Progenitor Cells Confers HIV-1 Resistance In Vivo
- (2017) Lei Xu et al. MOLECULAR THERAPY
- Modified gold-based siRNA nanotherapeutics for targeted therapy of triple-negative breast cancer
- (2017) Reza Shahbazi et al. Nanomedicine
- Targeting a CAR to the TRAC locus with CRISPR/Cas9 enhances tumour rejection
- (2017) Justin Eyquem et al. NATURE
- Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing
- (2017) Hao Yin et al. NATURE BIOTECHNOLOGY
- Refining strategies to translate genome editing to the clinic
- (2017) Tatjana I Cornu et al. NATURE MEDICINE
- Non-homologous DNA end joining and alternative pathways to double-strand break repair
- (2017) Howard H. Y. Chang et al. NATURE REVIEWS MOLECULAR CELL BIOLOGY
- Anti-inflammatory, analgesic and anti-tumor properties of gold nanoparticles
- (2017) Raimundo Fernandes de Araújo et al. Pharmacological Reports
- A distinct hematopoietic stem cell population for rapid multilineage engraftment in nonhuman primates
- (2017) Stefan Radtke et al. Science Translational Medicine
- CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease
- (2017) Suk See De Ravin et al. Science Translational Medicine
- Synthetically modified guide RNA and donor DNA are a versatile platform for CRISPR-Cas9 engineering
- (2017) Kunwoo Lee et al. eLife
- Cas9, Cpf1 and C2c1/2/3―What's next?
- (2017) Shota Nakade et al. Bioengineered
- In Vivo Murine-Matured Human CD3 + Cells as a Preclinical Model for T Cell-Based Immunotherapies
- (2017) Kevin G. Haworth et al. Molecular Therapy-Methods & Clinical Development
- Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair
- (2017) Kunwoo Lee et al. Nature Biomedical Engineering
- CRISPR/Cas9 for Human Genome Engineering and Disease Research
- (2016) Xin Xiong et al. Annual Review of Genomics and Human Genetics
- Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency
- (2016) Maria Pia Cicalese et al. BLOOD
- Crystal Structure of Cpf1 in Complex with Guide RNA and Target DNA
- (2016) Takashi Yamano et al. CELL
- Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial
- (2016) Maria Sessa et al. LANCET
- Functionalized gold nanoparticles manifested as potent carriers for nucleolar targeting
- (2016) Reza Shahbazi et al. NANOTECHNOLOGY
- CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells
- (2016) Daniel P. Dever et al. NATURE
- A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition
- (2016) Elizabeth A Traxler et al. NATURE MEDICINE
- Semi-automated closed system manufacturing of lentivirus gene-modified haematopoietic stem cells for gene therapy
- (2016) Jennifer E. Adair et al. Nature Communications
- Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System
- (2015) Bernd Zetsche et al. CELL
- Challenges in CRISPR/CAS9 Delivery: Potential Roles of Nonviral Vectors
- (2015) Ling Li et al. HUMAN GENE THERAPY
- Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome
- (2015) Salima Hacein-Bey Abina et al. JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
- Distinct routes of lineage development reshape the human blood hierarchy across ontogeny
- (2015) F. Notta et al. SCIENCE
- Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases
- (2014) S. Bae et al. BIOINFORMATICS
- Gold Nanoparticles for Nucleic Acid Delivery
- (2014) Ya Ding et al. MOLECULAR THERAPY
- A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency
- (2014) Salima Hacein-Bey-Abina et al. NEW ENGLAND JOURNAL OF MEDICINE
- Easy quantitative assessment of genome editing by sequence trace decomposition
- (2014) Eva K. Brinkman et al. NUCLEIC ACIDS RESEARCH
- PEAR: a fast and accurate Illumina Paired-End reAd mergeR
- (2013) J. Zhang et al. BIOINFORMATICS
- The Possible “Proton Sponge ” Effect of Polyethylenimine (PEI) Does Not Include Change in Lysosomal pH
- (2012) Rikke V Benjaminsen et al. MOLECULAR THERAPY
- Fetal hemoglobin in sickle cell anemia
- (2011) I. Akinsheye et al. BLOOD
- Toxicity and cellular uptake of gold nanoparticles: what we have learned so far?
- (2010) Alaaldin M. Alkilany et al. JOURNAL OF NANOPARTICLE RESEARCH
- Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease
- (2010) Stefan Stein et al. NATURE MEDICINE
- CCR5: From Natural Resistance to a New Anti-HIV Strategy
- (2010) Lucia Lopalco Viruses-Basel
- Cytotoxicity of Nanoparticles
- (2007) Nastassja Lewinski et al. Small
Publish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn MoreCreate your own webinar
Interested in hosting your own webinar? Check the schedule and propose your idea to the Peeref Content Team.
Create Now