Related references
Note: Only part of the references are listed.Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A
G. N. Nguyen et al.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS (2017)
Stable In Vivo Transgene Expression in Endothelial Cells with Helper-Dependent Adenovirus: Roles of Promoter and Interleukin-10
Nagadhara Dronadula et al.
HUMAN GENE THERAPY (2017)
Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy
Hildegund C. J. Ertl et al.
HUMAN GENE THERAPY (2017)
Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models
Randy J. Chandler et al.
HUMAN GENE THERAPY (2017)
Microfluidic Transduction Harnesses Mass Transport Principles to Enhance Gene Transfer Efficiency
Reginald Tran et al.
MOLECULAR THERAPY (2017)
Identification of liver-specific enhancer-promoter activity in the 3′ untranslated region of the wild-type AAV2 genome
Grant J. Logan et al.
NATURE GENETICS (2017)
被撤回的出版物: Unexpected mutations after CRISPR-Cas9 editing in vivo (Retracted article. See vol. 14, pg. 547, 2017)
Kellie A. Schaefer et al.
NATURE METHODS (2017)
The contribution of homology arms to nuclease-assisted genome engineering
Oliver Baker et al.
NUCLEIC ACIDS RESEARCH (2017)
CRISPR/Cas9 targeting events cause complex deletions and insertions at 17 sites in the mouse genome
Ha Youn Shin et al.
NATURE COMMUNICATIONS (2017)
Neonatal Gene Therapy for Hemophilia B by a Novel Adenovirus Vector Showing Reduced Leaky Expression of Viral Genes
Shunsuke Iizuka et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2017)
Homology-mediated end joining-based targeted integration using CRISPR/Cas9
Xuan Yao et al.
CELL RESEARCH (2017)
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
Christopher E. Nelson et al.
SCIENCE (2016)
In vivo transduction of primitive mobilized hematopoietic stem cells after intravenous injection of integrating adenovirus vectors
Maximilian Richter et al.
BLOOD (2016)
CRISPR/Cas9-mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse
Yuting Guan et al.
EMBO MOLECULAR MEDICINE (2016)
High Efficiency CRISPR/Cas9-mediated Gene Editing in Primary Human T-cells Using Mutant Adenoviral E4orf6/E1b55k Helper Proteins
Kamila S. Gwiazda et al.
MOLECULAR THERAPY (2016)
A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice
Yang Yang et al.
NATURE BIOTECHNOLOGY (2016)
Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo
Hao Yin et al.
NATURE BIOTECHNOLOGY (2016)
A multifunctional AAV-CRISPR-Cas9 and its host response
Wei Leong Chew et al.
NATURE METHODS (2016)
Adenovirus sensing by the immune system
Svetlana Atasheva et al.
CURRENT OPINION IN VIROLOGY (2016)
LiPS-A3S, a human genomic site for robust expression of inserted transgenes
Andriana G Kotini et al.
Molecular Therapy-Nucleic Acids (2016)
Systematic quantification of HDR and NHEJ reveals effects of locus, nuclease, and cell type on genome-editing
Yuichiro Miyaoka et al.
SCIENTIFIC REPORTS (2016)
In vivo genome editing of the albumin locus as a platform for protein replacement therapy
Rajiv Sharma et al.
BLOOD (2015)
Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing Adeno-Associated Virus Clinical Trial
Paul E. Monahan et al.
HUMAN GENE THERAPY (2015)
Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses
Dan Wang et al.
HUMAN GENE THERAPY (2015)
Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining
Takeshi Maruyama et al.
NATURE BIOTECHNOLOGY (2015)
Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells
Van Trung Chu et al.
NATURE BIOTECHNOLOGY (2015)
Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas
Jean-Charles Nault et al.
NATURE GENETICS (2015)
Generation of a stable packaging cell line producing high-titer PPT-deleted integration-deficient lentiviral vectors
Peirong Hu et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2015)
Promoter less gene targeting without nucleases ameliorates haemophilia B in mice
A. Barzel et al.
NATURE (2015)
A conditional knockout mouse model reveals endothelial cells as the principal and possibly exclusive source of plasma factor VIII
Scot A. Fahs et al.
BLOOD (2014)
Efficient gene targeting of the Rosa26 locus in mouse zygotes using TALE nucleases
Petr Kasparek et al.
FEBS LETTERS (2014)
Adenovirus: The First Effective In Vivo Gene Delivery Vector
Ronald G. Crystal
HUMAN GENE THERAPY (2014)
Uncovering and Dissecting the Genotoxicity of Self-inactivating Lentiviral Vectors In Vivo
Daniela Cesana et al.
MOLECULAR THERAPY (2014)
Adenoviral vector DNA for accurate genome editing with engineered nucleases
Maarten Holkers et al.
NATURE METHODS (2014)
Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
A. C. Nathwani et al.
NEW ENGLAND JOURNAL OF MEDICINE (2014)
Gene Therapy for Wiskott-Aldrich Syndrome-Long-Term Efficacy and Genotoxicity
Christian Joerg Braun et al.
SCIENCE TRANSLATIONAL MEDICINE (2014)
Microhomology-mediated end-joining-dependent integration of donor DNA in cells and animals using TALENs and CRISPR/Cas9
Shota Nakade et al.
NATURE COMMUNICATIONS (2014)
Retargeting of gene expression using endothelium specific hexon modified adenoviral vector
Sergey A. Kaliberov et al.
VIROLOGY (2013)
Genomic cis-acting Sequences Improve Expression and Establishment of a Nonviral Vector
Claudia Hagedorn et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2013)
Safe harbours for the integration of new DNA in the human genome
Michel Sadelain et al.
NATURE REVIEWS CANCER (2012)
Fate tracing of mature hepatocytes in mouse liver homeostasis and regeneration
Yann Malato et al.
JOURNAL OF CLINICAL INVESTIGATION (2011)
In vivo genome editing restores haemostasis in a mouse model of haemophilia
Hojun Li et al.
NATURE (2011)
A Comparison of Exogenous Promoter Activity at the ROSA26 Locus Using a PhiC31 Integrase Mediated Cassette Exchange Approach in Mouse ES Cells
Chiann-mun Chen et al.
PLOS ONE (2011)
Chromosomal Integration of Adenoviral Vector DNA In Vivo
Sam Laurel Stephen et al.
JOURNAL OF VIROLOGY (2010)
Adeno-associated virus integration: virus versus vector
R. H. Smith
GENE THERAPY (2008)
Adeno-associated virus vector Genomes persist as episomal chromatin in primate muscle
Magalie Penaud-Budloo et al.
JOURNAL OF VIROLOGY (2008)
High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR)
Schmidt Manfred et al.
NATURE METHODS (2007)
Identification and targeting of the ROSA26 locus in human embryonic stem cells
Stefan Irion et al.
NATURE BIOTECHNOLOGY (2007)
AAV vector integration sites in mouse hepatocellular carcinoma
Anthony Donsante et al.
SCIENCE (2007)
Histone modifications are associated with the persistence or silencing of vector-mediated transgene expression in vivo
Efren Riu et al.
MOLECULAR THERAPY (2007)
Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response
CS Manno et al.
NATURE MEDICINE (2006)
Long-term transgene expression in proliferating cells mediated by episomally maintained high-capacity adenovirus vectors
F Kreppel et al.
JOURNAL OF VIROLOGY (2004)
Episomal persistence of recombinant adenoviral vector Genomes during the cell cycle in vivo
A Ehrhardt et al.
JOURNAL OF VIROLOGY (2003)
Insulation from viral transcriptional regulatory elements improves inducible transgene expression from adenovirus vectors in vitro and in vivo
DS Steinwaerder et al.
GENE THERAPY (2000)
Molecular mechanism for silencing virally transduced genes involves histone deacetylation and chromatin condensation
WY Chen et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2000)
Share Paper
