Gene editing of PKLR gene in human hematopoietic progenitors through 5’ and 3’ UTR modified TALEN mRNA
出版年份 2019 全文链接
标题
Gene editing of PKLR gene in human hematopoietic progenitors through 5’ and 3’ UTR modified TALEN mRNA
作者
关键词
-
出版物
PLoS One
Volume 14, Issue 10, Pages e0223775
出版商
Public Library of Science (PLoS)
发表日期
2019-10-17
DOI
10.1371/journal.pone.0223775
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。- Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
- (2018) Alexis A. Thompson et al. NEW ENGLAND JOURNAL OF MEDICINE
- Gene therapy comes of age
- (2018) Cynthia E. Dunbar et al. SCIENCE
- Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome
- (2018) Caroline Y. Kuo et al. Cell Reports
- Worldwide study of hematopoietic allogeneic stem cell transplantation in pyruvate kinase deficiency
- (2017) Stephanie van Straaten et al. HAEMATOLOGICA
- Highly efficient genome editing of human hematopoietic stem cells via a nano-silicon-blade delivery approach
- (2017) Yuan Ma et al. Integrative Biology
- Autologous Induced Stem-Cell–Derived Retinal Cells for Macular Degeneration
- (2017) Michiko Mandai et al. NEW ENGLAND JOURNAL OF MEDICINE
- Gene Therapy in a Patient with Sickle Cell Disease
- (2017) Jean-Antoine Ribeil et al. NEW ENGLAND JOURNAL OF 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
- Engineering the haemogenic niche mitigates endogenous inhibitory signals and controls pluripotent stem cell-derived blood emergence
- (2017) Nafees Rahman et al. Nature Communications
- Worldwide study of hematopoietic allogeneic stem cell transplantation in pyruvate kinase deficiency
- (2017) Stephanie van Straaten et al. HAEMATOLOGICA
- Combining Single Strand Oligodeoxynucleotides and CRISPR/Cas9 to Correct Gene Mutations in β-Thalassemia-induced Pluripotent Stem Cells
- (2016) Xiaohua Niu et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells
- (2016) Megan D Hoban et al. MOLECULAR THERAPY
- Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency
- (2016) Maria Garcia-Gomez et al. MOLECULAR THERAPY
- 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
- Genome editing comes of age
- (2016) Jin-Soo Kim Nature Protocols
- Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia
- (2016) Lin Ye et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells
- (2016) M. A. DeWitt et al. Science Translational Medicine
- Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells
- (2015) M. D. Hoban et al. BLOOD
- Functional footprinting of regulatory DNA
- (2015) Jeff Vierstra et al. NATURE METHODS
- 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
- Both TALENs and CRISPR/Cas9 directly target the HBB IVS2–654 (C > T) mutation in β-thalassemia-derived iPSCs
- (2015) Peng Xu et al. Scientific Reports
- Modeling Human Severe Combined Immunodeficiency and Correction by CRISPR/Cas9-Enhanced Gene Targeting
- (2015) Chia-Wei Chang et al. Cell Reports
- Generation of a High Number of Healthy Erythroid Cells from Gene-Edited Pyruvate Kinase Deficiency Patient-Specific Induced Pluripotent Stem Cells
- (2015) Zita Garate et al. Stem Cell Reports
- Targeted gene therapy and cell reprogramming in Fanconi anemia
- (2014) P. Rio et al. EMBO Molecular Medicine
- Targeted genome editing in human repopulating haematopoietic stem cells
- (2014) Pietro Genovese et al. NATURE
- CRISPR-Cas systems for editing, regulating and targeting genomes
- (2014) Jeffry D Sander et al. NATURE BIOTECHNOLOGY
- Pyrimidoindole derivatives are agonists of human hematopoietic stem cell self-renewal
- (2014) I. Fares et al. SCIENCE
- A Viral RNA Structural Element Alters Host Recognition of Nonself RNA
- (2014) J. L. Hyde et al. SCIENCE
- Prostaglandin-modulated umbilical cord blood hematopoietic stem cell transplantation
- (2013) C. Cutler et al. BLOOD
- Ribosomal and hematopoietic defects in induced pluripotent stem cells derived from Diamond Blackfan anemia patients
- (2013) L. Garcon et al. BLOOD
- Induction of Multipotential Hematopoietic Progenitors from Human Pluripotent Stem Cells via Respecification of Lineage-Restricted Precursors
- (2013) Sergei Doulatov et al. Cell Stem Cell
- Nucleofection of Expression Vectors Induces a Robust Interferon Response and Inhibition of Cell Proliferation
- (2013) Sandra Huerfano et al. DNA AND CELL BIOLOGY
- Generation of Engraftable Hematopoietic Stem Cells From Induced Pluripotent Stem Cells by Way of Teratoma Formation
- (2013) Nao Suzuki et al. MOLECULAR THERAPY
- Zinc-finger nuclease-mediated correction of -thalassemia in iPS cells
- (2012) C.-J. Chang et al. BLOOD
- Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting
- (2011) J. Zou et al. BLOOD
- Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease
- (2011) J. Zou et al. BLOOD
- Pulse exposure of haematopoietic grafts to prostaglandin E2in vitro facilitates engraftment and recovery
- (2011) L. M. Pelus et al. CELL PROLIFERATION
- Prostaglandin E2 Enhances Human Cord Blood Stem Cell Xenotransplants and Shows Long-Term Safety in Preclinical Nonhuman Primate Transplant Models
- (2011) Wolfram Goessling et al. Cell Stem Cell
- In Situ Genetic Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells Using Engineered Zinc Finger Nucleases
- (2011) Vittorio Sebastiano et al. STEM CELLS
- Quantification of lentiviral vector copy numbers in individual hematopoietic colony-forming cells shows vector dose-dependent effects on the frequency and level of transduction
- (2010) S Charrier et al. GENE THERAPY
- Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
- (2010) Marina Cavazzana-Calvo et al. NATURE
- Aryl Hydrocarbon Receptor Antagonists Promote the Expansion of Human Hematopoietic Stem Cells
- (2010) Anthony E. Boitano et al. SCIENCE
- Prostaglandin E2 enhances hematopoietic stem cell homing, survival, and proliferation
- (2009) J. Hoggatt et al. BLOOD
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