Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing
出版年份 2019 全文链接
标题
Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing
作者
关键词
-
出版物
Nature Communications
Volume 10, Issue 1, Pages -
出版商
Springer Science and Business Media LLC
发表日期
2019-08-07
DOI
10.1038/s41467-019-11454-9
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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。- Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1
- (2019) Ewelina Mamcarz et al. NEW ENGLAND JOURNAL OF MEDICINE
- CRISPR-Cas9: A cornerstone for the evolution of precision medicine
- (2018) Sleiman Razzouk ANNALS OF HUMAN GENETICS
- Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis
- (2018) Ema Robinson et al. MOLECULAR THERAPY
- A highly specific SpCas9 variant is identified by in vivo screening in yeast
- (2018) Antonio Casini et al. NATURE BIOTECHNOLOGY
- Cas9 versus Cas12a/Cpf1: Structure-function comparisons and implications for genome editing
- (2018) Daan C. Swarts et al. Wiley Interdisciplinary Reviews-RNA
- VSV-G-Enveloped Vesicles for Traceless Delivery of CRISPR-Cas9
- (2018) Claudia Montagna et al. Molecular Therapy-Nucleic Acids
- CRISPR-Cas guides the future of genetic engineering
- (2018) Gavin J. Knott et al. SCIENCE
- Predictable and precise template-free CRISPR editing of pathogenic variants
- (2018) Max W. Shen et al. NATURE
- In vitro Methods for the Development and Analysis of Human Primary Airway Epithelia
- (2018) Ambra Gianotti et al. Frontiers in Pharmacology
- Human Primary Epithelial Cell Models: Promising Tools in the Era of Cystic Fibrosis Personalized Medicine
- (2018) Nikhil T. Awatade et al. Frontiers in Pharmacology
- Genetic Modification of the Lung Directed Toward Treatment of Human Disease
- (2017) Dolan Sondhi et al. HUMAN GENE THERAPY
- CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10
- (2017) Guo-Xiang Ruan et al. MOLECULAR THERAPY
- Engineered Cpf1 variants with altered PAM specificities
- (2017) Linyi Gao et al. NATURE BIOTECHNOLOGY
- Roadmap for an early gene therapy for cystic fibrosis airway disease
- (2017) Marianne S. Carlon et al. PRENATAL DIAGNOSIS
- Hit and go CAS9 delivered through a lentiviral based self-limiting circuit
- (2017) Gianluca Petris et al. Nature Communications
- Inherited determinants of early recurrent somatic mutations in prostate cancer
- (2017) Alessandro Romanel et al. Nature Communications
- rAAV-CFTRΔR Rescues the Cystic Fibrosis Phenotype in Human Intestinal Organoids and Cystic Fibrosis Mice
- (2016) Dragana Vidović et al. AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
- DNA Repair Profiling Reveals Nonrandom Outcomes at Cas9-Mediated Breaks
- (2016) Megan van Overbeek et al. MOLECULAR CELL
- An essential receptor for adeno-associated virus infection
- (2016) S. Pillay et al. NATURE
- Analyzing CRISPR genome-editing experiments with CRISPResso
- (2016) Luca Pinello et al. NATURE BIOTECHNOLOGY
- Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9
- (2016) John G Doench et al. NATURE BIOTECHNOLOGY
- Genome-wide specificities of CRISPR-Cas Cpf1 nucleases in human cells
- (2016) Benjamin P Kleinstiver et al. NATURE BIOTECHNOLOGY
- Genome-wide analysis reveals specificities of Cpf1 endonucleases in human cells
- (2016) Daesik Kim et al. NATURE BIOTECHNOLOGY
- Organoids as an in vitro model of human development and disease
- (2016) Aliya Fatehullah et al. NATURE CELL BIOLOGY
- Genetic medicines for CF: Hype versus reality
- (2016) Eric W.F.W. Alton et al. PEDIATRIC PULMONOLOGY
- Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis
- (2016) Johanna F. Dekkers et al. Science Translational Medicine
- Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis
- (2016) Eric W F W Alton et al. THORAX
- Targeted Integration of a Super-Exon into the CFTR Locus Leads to Functional Correction of a Cystic Fibrosis Cell Line Model
- (2016) Christien Bednarski et al. PLoS One
- Engineered tobacco etch virus (TEV) protease active in the secretory pathway of mammalian cells
- (2015) Francesca Cesaratto et al. JOURNAL OF BIOTECHNOLOGY
- Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium
- (2015) Nicole Ali McNeer et al. Nature Communications
- Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
- (2015) Eric W F W Alton et al. Lancet Respiratory Medicine
- Immunological Ignorance Allows Long-Term Gene Expression After Perinatal Recombinant Adeno-Associated Virus-Mediated Gene Transfer to Murine Airways
- (2014) Marianne S. Carlon et al. HUMAN GENE THERAPY
- Reduction of HIV-1 Infectivity through Endoplasmic Reticulum-Associated Degradation-Mediated Env Depletion
- (2014) Antonio Casini et al. JOURNAL OF VIROLOGY
- Targeted genome editing in human repopulating haematopoietic stem cells
- (2014) Pietro Genovese et al. NATURE
- GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases
- (2014) Shengdar Q Tsai et al. NATURE BIOTECHNOLOGY
- Genome-wide detection of DNA double-stranded breaks induced by engineered nucleases
- (2014) Richard L Frock et al. NATURE BIOTECHNOLOGY
- Cystic fibrosis genetics: from molecular understanding to clinical application
- (2014) Garry R. Cutting NATURE REVIEWS GENETICS
- Easy quantitative assessment of genome editing by sequence trace decomposition
- (2014) Eva K. Brinkman et al. NUCLEIC ACIDS RESEARCH
- Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients
- (2013) Gerald Schwank et al. Cell Stem Cell
- A functional CFTR assay using primary cystic fibrosis intestinal organoids
- (2013) Johanna F Dekkers et al. NATURE MEDICINE
- Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells
- (2013) Ophir Shalem et al. SCIENCE
- An exon-specific U1 small nuclear RNA (snRNA) strategy to correct splicing defects
- (2012) Eugenio Fernandez Alanis et al. HUMAN MOLECULAR GENETICS
- Virus-mediated gene delivery for human gene therapy
- (2012) Mauro Giacca et al. JOURNAL OF CONTROLLED RELEASE
- Extensive Methylation of Promoter Sequences Silences Lentiviral Transgene Expression During Stem Cell Differentiation In Vivo
- (2012) Friederike Herbst et al. MOLECULAR THERAPY
- Long-term Expansion of Epithelial Organoids From Human Colon, Adenoma, Adenocarcinoma, and Barrett's Epithelium
- (2011) Toshiro Sato et al. GASTROENTEROLOGY
- Bioinformatics enrichment tools: paths toward the comprehensive functional analysis of large gene lists
- (2008) Da Wei Huang et al. NUCLEIC ACIDS RESEARCH
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