CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice
出版年份 2017 全文链接
标题
CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice
作者
关键词
-
出版物
Scientific Reports
Volume 7, Issue 1, Pages -
出版商
Springer Nature
发表日期
2017-06-19
DOI
10.1038/s41598-017-04625-5
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。- AAV Infection: Protection from Cancer
- (2017) Arun Srivastava et al. HUMAN GENE THERAPY
- CRISPR/Cas9‐mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse
- (2016) Yuting Guan et al. EMBO Molecular Medicine
- In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration
- (2016) Keiichiro Suzuki et al. NATURE
- Correction of the auditory phenotype in C57BL/6N mice via CRISPR/Cas9-mediated homology directed repair
- (2016) Joffrey Mianné et al. Genome Medicine
- CRISPR-Cas9 for in vivo Gene Therapy: Promise and Hurdles
- (2016) Wei-Jing Dai et al. Molecular Therapy-Nucleic Acids
- CRISPR-Cas: New Tools for Genetic Manipulations from Bacterial Immunity Systems
- (2015) Wenyan Jiang et al. Annual Review of Microbiology
- In vivo genome editing of the albumin locus as a platform for protein replacement therapy
- (2015) R. Sharma et al. BLOOD
- Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System
- (2015) Bernd Zetsche et al. CELL
- Adeno-associated virus serotypes for gene therapeutics
- (2015) Leszek Lisowski et al. CURRENT OPINION IN PHARMACOLOGY
- Inhibition of nonhomologous end joining to increase the specificity of CRISPR/Cas9 genome editing
- (2015) Supriya V. Vartak et al. FEBS Journal
- New approaches to gene and cell therapy for hemophilia
- (2015) T. Ohmori et al. JOURNAL OF THROMBOSIS AND HAEMOSTASIS
- Don’t edit the human germ line
- (2015) Edward Lanphier et al. NATURE
- In vivo genome editing using Staphylococcus aureus Cas9
- (2015) F. Ann Ran 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
- 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
- An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia
- (2015) Alfica Sehgal et al. NATURE MEDICINE
- Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy
- (2015) C. Long et al. SCIENCE
- In vivo gene editing in dystrophic mouse muscle and muscle stem cells
- (2015) M. Tabebordbar et al. SCIENCE
- In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
- (2015) C. E. Nelson et al. SCIENCE
- Development and Applications of CRISPR-Cas9 for Genome Engineering
- (2014) Patrick D. Hsu et al. CELL
- CRISPR/Cas9 for genome editing: progress, implications and challenges
- (2014) F. Zhang et al. HUMAN MOLECULAR GENETICS
- Mechanisms and principles of homology search during recombination
- (2014) Jörg Renkawitz et al. NATURE REVIEWS MOLECULAR CELL BIOLOGY
- Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA
- (2014) C. Long et al. SCIENCE
- The new frontier of genome engineering with CRISPR-Cas9
- (2014) J. A. Doudna et al. SCIENCE
- Robust ZFN-mediated genome editing in adult hemophilic mice
- (2013) X. M. Anguela et al. BLOOD
- The prevalence of neutralizing antibodies against adeno-associated virus capsids is reduced in young Japanese individuals
- (2013) Jun Mimuro et al. JOURNAL OF MEDICAL VIROLOGY
- High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity
- (2013) Vikram Pattanayak et al. NATURE BIOTECHNOLOGY
- High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells
- (2013) Yanfang Fu et al. NATURE BIOTECHNOLOGY
- Cas9 as a versatile tool for engineering biology
- (2013) Prashant Mali et al. NATURE METHODS
- ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering
- (2013) Thomas Gaj et al. TRENDS IN BIOTECHNOLOGY
- The CDC Hemophilia B mutation project mutation list: a new online resource
- (2013) Tengguo Li et al. Molecular Genetics & Genomic Medicine
- The AAV Vector Toolkit: Poised at the Clinical Crossroads
- (2012) Aravind Asokan et al. MOLECULAR THERAPY
- Minimizing the Inhibitory Effect of Neutralizing Antibody for Efficient Gene Expression in the Liver With Adeno-associated Virus 8 Vectors
- (2012) Jun Mimuro et al. MOLECULAR THERAPY
- Hepatic Gene Transfer in Neonatal Mice by Adeno-Associated Virus Serotype 8 Vector
- (2011) Lili Wang et al. HUMAN GENE THERAPY
- In vivo genome editing restores haemostasis in a mouse model of haemophilia
- (2011) Hojun Li et al. NATURE
- A modified thrombin generation test for investigating very low levels of factor VIII activity in hemophilia A
- (2009) Tomoko Matsumoto et al. INTERNATIONAL JOURNAL OF HEMATOLOGY
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 MoreAdd your recorded webinar
Do you already have a recorded webinar? Grow your audience and get more views by easily listing your recording on Peeref.
Upload Now