标题
Somatic genome editing with CRISPR/Cas9 generates and corrects a metabolic disease
作者
关键词
-
出版物
Scientific Reports
Volume 7, Issue 1, Pages -
出版商
Springer Nature
发表日期
2017-03-16
DOI
10.1038/srep44624
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。- CRISPR-Cas9 Targeting ofPCSK9in Human Hepatocytes In Vivo—Brief ReportSignificance
- (2016) Xiao Wang et al. ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY
- Inhibition of apolipoprotein B synthesis stimulates endoplasmic reticulum autophagy that prevents steatosis
- (2016) Donna M. Conlon et al. JOURNAL OF CLINICAL INVESTIGATION
- The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian Cells
- (2016) Ciaran M Lee et al. MOLECULAR THERAPY
- Recombinant AAV Integration Is Not Associated With Hepatic Genotoxicity in Nonhuman Primates and Patients
- (2016) Irene Gil-Farina et al. MOLECULAR THERAPY
- Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo
- (2016) Hao Yin et al. NATURE BIOTECHNOLOGY
- Probing the impact of chromatin conformation on genome editing tools
- (2016) Xiaoyu Chen et al. NUCLEIC ACIDS RESEARCH
- Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia
- (2016) Francis P. Pankowicz et al. Nature Communications
- Mipomersen and lomitapide: Two new drugs for the treatment of homozygous familial hypercholesterolemia
- (2015) Ioanna Gouni-Berthold et al. ATHEROSCLEROSIS SUPPLEMENTS
- Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy
- (2015) Randy J. Chandler et al. JOURNAL OF CLINICAL INVESTIGATION
- In vivo genome editing using Staphylococcus aureus Cas9
- (2015) F. Ann Ran et al. NATURE
- Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas
- (2015) Jean-Charles Nault et al. NATURE GENETICS
- Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model
- (2015) Beatrice Bissig-Choisat et al. Nature Communications
- Genetic manipulation of the ApoF/Stat2 locus supports an important role for type I interferon signaling in atherosclerosis
- (2014) William R. Lagor et al. ATHEROSCLEROSIS
- Clinical experience of lomitapide therapy in patients with homozygous familial hypercholesterolaemia
- (2014) Marina Cuchel et al. ATHEROSCLEROSIS SUPPLEMENTS
- CRISPR/Cas9-mediated genome engineering: An adeno-associated viral (AAV) vector toolbox
- (2014) Elena Senís et al. Biotechnology Journal
- CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling
- (2014) Randall J. Platt et al. CELL
- E-CRISP: fast CRISPR target site identification
- (2014) Florian Heigwer et al. NATURE METHODS
- CRISPR/Cas9 systems have off-target activity with insertions or deletions between target DNA and guide RNA sequences
- (2014) Y. Lin et al. NUCLEIC ACIDS RESEARCH
- COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites
- (2014) Thomas J Cradick et al. Molecular Therapy-Nucleic Acids
- Bloomsbury report on mouse embryo phenotyping: recommendations from the IMPC workshop on embryonic lethal screening
- (2013) D. Adams et al. Disease Models & Mechanisms
- Therapeutic expression of hairpins targeting apolipoprotein B100 induces phenotypic and transcriptome changes in murine liver
- (2013) P Maczuga et al. GENE THERAPY
- Lipid overloading during liver regeneration causes delayed hepatocyte DNA replication by increasing ER stress in mice with simple hepatic steatosis
- (2013) Mina Hamano et al. JOURNAL OF GASTROENTEROLOGY
- Delivery materials for siRNA therapeutics
- (2013) Rosemary Kanasty et al. NATURE MATERIALS
- Genome engineering using the CRISPR-Cas9 system
- (2013) F Ann Ran et al. Nature Protocols
- RNA-Guided Human Genome Engineering via Cas9
- (2013) P. Mali et al. SCIENCE
- Multiplex Genome Engineering Using CRISPR/Cas Systems
- (2013) L. Cong et al. SCIENCE
- Antisense Technology: An Emerging Platform for Cardiovascular Disease Therapeutics
- (2013) Richard G. Lee et al. Journal of Cardiovascular Translational Research
- Adeno-Associated Virus Serotype 8 Gene Therapy Leads to Significant Lowering of Plasma Cholesterol Levels in Humanized Mouse Models of Homozygous and Heterozygous Familial Hypercholesterolemia
- (2012) Sadik H. Kassim et al. HUMAN GENE THERAPY
- NIH Image to ImageJ: 25 years of image analysis
- (2012) Caroline A Schneider et al. NATURE METHODS
- Locked Nucleic Acids: Promising Nucleic Acid Analogs for Therapeutic Applications
- (2010) Rakesh N. Veedu et al. CHEMISTRY & BIODIVERSITY
- Regulation of Bile Acid Synthesis by Fat-soluble Vitamins A and D
- (2010) Daniel R. Schmidt et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- The burden of chronic venous diseases is unknown
- (2010) J-J Guex PHLEBOLOGY
- High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency
- (2009) E Ayuso et al. GENE THERAPY
- Contrasting features of urea cycle disorders in human patients and knockout mouse models
- (2008) Joshua L. Deignan et al. MOLECULAR GENETICS AND METABOLISM
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