Ex vivo and in vivo genome editing: a regulatory scientific framework from early development to clinical implementation
出版年份 2017 全文链接
标题
Ex vivo and in vivo genome editing: a regulatory scientific framework from early development to clinical implementation
作者
关键词
-
出版物
Regenerative Medicine
Volume 12, Issue 8, Pages 1015-1030
出版商
Future Medicine Ltd
发表日期
2017-12-15
DOI
10.2217/rme-2017-0095
参考文献
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注意:仅列出部分参考文献,下载原文获取全部文献信息。- Integration of a CD19 CAR into the TCR Alpha Chain Locus Streamlines Production of Allogeneic Gene-Edited CAR T Cells
- (2017) Daniel T. MacLeod et al. MOLECULAR THERAPY
- Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery
- (2017) Thomas Gaj et al. NUCLEIC ACIDS RESEARCH
- CRISPR-targeted genome editing of mesenchymal stem cell-derived therapies for type 1 diabetes: a path to clinical success?
- (2017) Dario Gerace et al. Stem Cell Research & Therapy
- Antiviral treatment strategies based on gene silencing and genome editing
- (2017) Roger Badia et al. Current Opinion in Virology
- CRISPR/Cas9-Mediated Genome Editing Corrects Dystrophin Mutation in Skeletal Muscle Stem Cells in a Mouse Model of Muscle Dystrophy
- (2017) Pei Zhu et al. Molecular Therapy-Nucleic Acids
- CRISPR-Cas9 mediated LAG-3 disruption in CAR-T cells
- (2017) Yongping Zhang et al. Frontiers of 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
- Programmable Genome Editing Tools and their Regulation for Efficient Genome Engineering
- (2017) Tuhin Kumar Guha et al. Computational and Structural Biotechnology Journal
- AAV Vectorization of DSB-mediated Gene Editing Technologies
- (2016) Rachel J. Moser et al. CURRENT GENE THERAPY
- Genome-editing technologies for gene correction of hemophilia
- (2016) Chul-Yong Park et al. HUMAN GENETICS
- Gene editing advance re-ignites debate on the merits and risks of animal to human transplantation
- (2016) R. K. F. Fung et al. INTERNAL MEDICINE JOURNAL
- Site-specific genome editing for correction of induced pluripotent stem cells derived from dominant dystrophic epidermolysis bullosa
- (2016) Satoru Shinkuma et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells
- (2015) M. D. Hoban et al. BLOOD
- In vivo genome editing of the albumin locus as a platform for protein replacement therapy
- (2015) R. Sharma et al. BLOOD
- Clinical Scale Zinc Finger Nuclease-mediated Gene Editing of PD-1 in Tumor Infiltrating Lymphocytes for the Treatment of Metastatic Melanoma
- (2015) Joal D Beane et al. MOLECULAR THERAPY
- Genome Editing Technologies: Defining a Path to Clinic
- (2015) Jacqueline Corrigan-Curay et al. MOLECULAR THERAPY
- Correction of Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients Through Genomic Excision of Exon 51 by Zinc Finger Nucleases
- (2015) David G Ousterout et al. MOLECULAR THERAPY
- Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells
- (2015) Ayal Hendel 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
- Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy
- (2015) David G. Ousterout et al. Nature Communications
- Functional Gene Correction for Cystic Fibrosis in Lung Epithelial Cells Generated from Patient iPSCs
- (2015) Amy L. Firth et al. Cell Reports
- 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
- Codon Swapping of Zinc Finger Nucleases Confers Expression in Primary Cells and In Vivo from a Single Lentiviral Vector
- (2014) Abarrategui-Pontes Cecilia 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
- Inactivation of the Human Papillomavirus E6 or E7 Gene in Cervical Carcinoma Cells by Using a Bacterial CRISPR/Cas RNA-Guided Endonuclease
- (2014) E. M. Kennedy et al. JOURNAL OF VIROLOGY
- Somatic Correction of Junctional Epidermolysis Bullosa by a Highly Recombinogenic AAV Variant
- (2014) Sandra P Melo et al. MOLECULAR THERAPY
- Targeted genome editing in human repopulating haematopoietic stem cells
- (2014) Pietro Genovese et al. NATURE
- mRNA-based therapeutics — developing a new class of drugs
- (2014) Ugur Sahin et al. NATURE REVIEWS DRUG DISCOVERY
- Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV
- (2014) Pablo Tebas et al. NEW ENGLAND JOURNAL OF MEDICINE
- AAV-Mediated Delivery of Zinc Finger Nucleases Targeting Hepatitis B Virus Inhibits Active Replication
- (2014) Nicholas D. Weber et al. PLoS One
- Novel lentiviral vectors with mutated reverse transcriptase for mRNA delivery of TALE nucleases
- (2014) Ulrike Mock et al. Scientific Reports
- Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients
- (2013) Gerald Schwank et al. Cell Stem Cell
- Inactivation of Hepatitis B Virus Replication in Cultured Cells and In Vivo with Engineered Transcription Activator-Like Effector Nucleases
- (2013) Kristie Bloom et al. MOLECULAR THERAPY
- TALEN-based Gene Correction for Epidermolysis Bullosa
- (2013) Mark J Osborn et al. MOLECULAR THERAPY
- Nuclease-mediated gene editing by homologous recombination of the human globin locus
- (2013) Richard A. Voit et al. NUCLEIC ACIDS RESEARCH
- Optimization of scarless human stem cell genome editing
- (2013) Luhan Yang et al. NUCLEIC ACIDS RESEARCH
- A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR
- (2012) H. Torikai et al. BLOOD
- mRNA as a Versatile Tool for Exogenous Protein Expression
- (2012) Andreas N. Kuhn et al. CURRENT GENE THERAPY
- Challenges in Vector and Trial Design Using Retroviral Vectors for Long-Term Gene Correction in Hematopoietic Stem Cell Gene Therapy
- (2012) Jacqueline Corrigan-Curay et al. MOLECULAR THERAPY
- Endgame: Glybera Finally Recommended for Approval as the First Gene Therapy Drug in the European Union
- (2012) Seppo Ylä-Herttuala MOLECULAR THERAPY
- Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer
- (2012) Elena Provasi et al. NATURE MEDICINE
- Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells
- (2012) Maarten Holkers et al. NUCLEIC ACIDS RESEARCH
- 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
- In vivo genome editing restores haemostasis in a mouse model of haemophilia
- (2011) Hojun Li et al. NATURE
- An unbiased genome-wide analysis of zinc-finger nuclease specificity
- (2011) Richard Gabriel et al. NATURE BIOTECHNOLOGY
- De novo-engineered transcription activator-like effector (TALE) hybrid nuclease with novel DNA binding specificity creates double-strand breaks
- (2011) M. M. Mahfouz et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- An Analysis of Pharmaceutical Experience with Decades of Rat Carcinogenicity Testing
- (2011) Frank D. Sistare et al. TOXICOLOGIC PATHOLOGY
- Recent Advances in Lentiviral Vector Development and Applications
- (2010) Janka Mátrai et al. MOLECULAR THERAPY
- Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo
- (2010) Nathalia Holt et al. NATURE BIOTECHNOLOGY
- Quantification of transforming capacity and cooperation of defined genetic alterations in myeloid malignancies
- (2009) Yildirim Dogan et al. EXPERIMENTAL HEMATOLOGY
- Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors
- (2009) J. Boch et al. SCIENCE
- Deconstructing Stem Cell Tumorigenicity: A Roadmap to Safe Regenerative Medicine
- (2009) Paul S. Knoepfler STEM CELLS
- Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases
- (2008) Elena E Perez et al. NATURE BIOTECHNOLOGY
- Replicative Senescence of Mesenchymal Stem Cells: A Continuous and Organized Process
- (2008) Wolfgang Wagner et al. PLoS One
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