标题
In utero CRISPR-mediated therapeutic editing of metabolic genes
作者
关键词
-
出版物
NATURE MEDICINE
Volume 24, Issue 10, Pages 1513-1518
出版商
Springer Nature America, Inc
发表日期
2018-10-04
DOI
10.1038/s41591-018-0184-6
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。- A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing
- (2018) Jonathan D. Finn et al. Cell Reports
- Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions
- (2017) Y Bill Kim et al. NATURE BIOTECHNOLOGY
- Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing
- (2017) Hao Yin et al. NATURE BIOTECHNOLOGY
- Genome-wide target specificities of CRISPR RNA-guided programmable deaminases
- (2017) Daesik Kim et al. NATURE BIOTECHNOLOGY
- Induction of Immune Tolerance to Foreign Protein via Adeno-Associated Viral Vector Gene Transfer in Mid-Gestation Fetal Sheep
- (2017) Marcus G. Davey et al. PLoS One
- CRISPR-Cas9 Targeting ofPCSK9in Human Hepatocytes In Vivo—Brief ReportSignificance
- (2016) Xiao Wang et al. ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY
- The Intravenous Route of Injection Optimizes Engraftment and Survival in the Murine Model of In Utero Hematopoietic Cell Transplantation
- (2016) Matthew M. Boelig et al. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
- Prenatal Diagnosis of Tyrosinemia Type 1 Using Next Generation Sequencing
- (2016) Maryam Rafati et al. Fetal and Pediatric Pathology
- Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
- (2016) Alexis C. Komor et al. NATURE
- A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice
- (2016) Yang Yang et al. NATURE BIOTECHNOLOGY
- Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo
- (2016) Hao Yin et al. NATURE BIOTECHNOLOGY
- Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia
- (2016) Francis P. Pankowicz et al. Nature Communications
- The Impact of Chromatin Dynamics on Cas9-Mediated Genome Editing in Human Cells
- (2016) René M. Daer et al. ACS Synthetic Biology
- Adenovirus-Mediated Somatic Genome Editing ofPtenby CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses
- (2015) Dan Wang et al. HUMAN GENE THERAPY
- In vivo genome editing using Staphylococcus aureus Cas9
- (2015) F. Ann Ran et al. NATURE
- Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing
- (2014) Qiurong Ding et al. CIRCULATION RESEARCH
- Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
- (2014) Hao Yin et al. NATURE BIOTECHNOLOGY
- Adenoviral Vector Immunity: Its Implications and Circumvention Strategies
- (2011) Yadvinder S. Ahi et al. CURRENT GENE THERAPY
- The developmental stage determines the distribution and duration of gene expression after early intra-amniotic gene transfer using lentiviral vectors
- (2009) M Endo et al. GENE THERAPY
- Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo
- (2009) Nicole K. Paulk et al. HEPATOLOGY
Find Funding. Review Successful Grants.
Explore over 25,000 new funding opportunities and over 6,000,000 successful grants.
ExplorePublish 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 More