RNA Editing as a Therapeutic Approach for Retinal Gene Therapy Requiring Long Coding Sequences
出版年份 2020 全文链接
标题
RNA Editing as a Therapeutic Approach for Retinal Gene Therapy Requiring Long Coding Sequences
作者
关键词
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出版物
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
Volume 21, Issue 3, Pages 777
出版商
MDPI AG
发表日期
2020-01-27
DOI
10.3390/ijms21030777
参考文献
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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。- Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10
- (2019) Morgan L. Maeder et al. NATURE MEDICINE
- High-Resolution Structure of Cas13b and Biochemical Characterization of RNA Targeting and Cleavage
- (2019) Ian M. Slaymaker et al. Cell Reports
- In vivo RNA editing of point mutations via RNA-guided adenosine deaminases
- (2019) Dhruva Katrekar et al. NATURE METHODS
- Precise RNA editing by recruiting endogenous ADARs with antisense oligonucleotides
- (2019) Tobias Merkle et al. NATURE BIOTECHNOLOGY
- In vivo genome editing rescues photoreceptor degeneration via a Cas9/RecA-mediated homology-directed repair pathway
- (2019) Yuan Cai et al. Science Advances
- Analysis and minimization of cellular RNA editing by DNA adenine base editors
- (2019) Holly A. Rees et al. Science Advances
- Programmable RNA-Guided RNA Effector Proteins Built from Human Parts
- (2019) Simone Rauch et al. CELL
- Programmable RNA editing by recruiting endogenous ADAR using engineered RNAs
- (2019) Liang Qu et al. NATURE BIOTECHNOLOGY
- A cytosine deaminase for programmable single-base RNA editing
- (2019) Omar O. Abudayyeh et al. SCIENCE
- Search-and-replace genome editing without double-strand breaks or donor DNA
- (2019) Andrew V. Anzalone et al. NATURE
- Transcriptome Engineering with RNA-Targeting Type VI-D CRISPR Effectors
- (2018) Silvana Konermann et al. CELL
- Molecular Mechanisms of RNA Targeting by Cas13-containing Type VI CRISPR–Cas Systems
- (2018) Mitchell R. O'Connell JOURNAL OF MOLECULAR BIOLOGY
- Cas13d Is a Compact RNA-Targeting Type VI CRISPR Effector Positively Modulated by a WYL-Domain-Containing Accessory Protein
- (2018) Winston X. Yan et al. MOLECULAR CELL
- Efficient and precise editing of endogenous transcripts with SNAP-tagged ADARs
- (2018) Paul Vogel et al. NATURE METHODS
- Clustered Regularly Interspaced Short Palindromic Repeats-Based Genome Surgery for the Treatment of Autosomal Dominant Retinitis Pigmentosa
- (2018) Yi-Ting Tsai et al. OPHTHALMOLOGY
- Gene therapy for visual loss: Opportunities and concerns
- (2018) Jia Hui Lee et al. PROGRESS IN RETINAL AND EYE RESEARCH
- RNA Binding and HEPN-Nuclease Activation Are Decoupled in CRISPR-Cas13a
- (2018) Akshay Tambe et al. Cell Reports
- Comparative Activity of Adenosine Deaminase Acting on RNA (ADARs) Isoforms for Correction of Genetic Code in Gene Therapy
- (2018) Thoufic Anam Azad et al. CURRENT GENE THERAPY
- Long-Term Effects of In Vivo Genome Editing in the Mouse Retina Using Campylobacter jejuni Cas9 Expressed via Adeno-Associated Virus
- (2018) Dong Hyun Jo et al. MOLECULAR THERAPY
- Base editing: precision chemistry on the genome and transcriptome of living cells
- (2018) Holly A. Rees et al. NATURE REVIEWS GENETICS
- ADAR RNA editing in human disease; more to it than meets the I
- (2017) Angela Gallo et al. HUMAN GENETICS
- Cas9/sgRNA selective targeting of the P23H Rhodopsin mutant allele for treating retinitis pigmentosa by intravitreal AAV9.PHP.B-based delivery
- (2017) Serena G Giannelli et al. HUMAN MOLECULAR GENETICS
- Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
- (2017) Stephen Russell et al. LANCET
- Cas13b Is a Type VI-B CRISPR-Associated RNA-Guided RNase Differentially Regulated by Accessory Proteins Csx27 and Csx28
- (2017) Aaron A. Smargon et al. MOLECULAR CELL
- Dynamic landscape and regulation of RNA editing in mammals
- (2017) Meng How Tan et al. NATURE
- RNA targeting with CRISPR–Cas13
- (2017) Omar O. Abudayyeh et al. NATURE
- Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions
- (2017) Y Bill Kim et al. NATURE BIOTECHNOLOGY
- Diversity and evolution of class 2 CRISPR–Cas systems
- (2017) Sergey Shmakov et al. NATURE REVIEWS MICROBIOLOGY
- ClinVar: improving access to variant interpretations and supporting evidence
- (2017) Melissa J Landrum et al. NUCLEIC ACIDS RESEARCH
- Clinically Focused Molecular Investigation of 1000 Consecutive Families with Inherited Retinal Disease
- (2017) Edwin M. Stone et al. OPHTHALMOLOGY
- Site-directed RNA repair of endogenous Mecp2 RNA in neurons
- (2017) John R. Sinnamon et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Unravelling the genetics of inherited retinal dystrophies: Past, present and future
- (2017) Suzanne Broadgate et al. PROGRESS IN RETINAL AND EYE RESEARCH
- Abundant off-target edits from site-directed RNA editing can be reduced by nuclear localization of the editing enzyme
- (2017) Isabel C. Vallecillo-Viejo et al. RNA Biology
- RNA editing with CRISPR-Cas13
- (2017) David B. T. Cox et al. SCIENCE
- Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice
- (2017) Wenhan Yu et al. Nature Communications
- Construction of a guide-RNA for site-directed RNA mutagenesis utilising intracellular A-to-I RNA editing
- (2017) Masatora Fukuda et al. Scientific Reports
- In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa
- (2016) Benjamin Bakondi et al. MOLECULAR THERAPY
- In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration
- (2016) Keiichiro Suzuki et al. NATURE
- Two distinct RNase activities of CRISPR-C2c2 enable guide-RNA processing and RNA detection
- (2016) Alexandra East-Seletsky et al. NATURE
- Structures of human ADAR2 bound to dsRNA reveal base-flipping mechanism and basis for site selectivity
- (2016) Melissa M Matthews et al. NATURE STRUCTURAL & MOLECULAR BIOLOGY
- Harnessing human ADAR2 for RNA repair – Recoding a PINK1 mutation rescues mitophagy
- (2016) Jacqueline Wettengel et al. NUCLEIC ACIDS RESEARCH
- An efficient system for selectively altering genetic information within mRNAs
- (2016) Maria Fernanda Montiel-González et al. NUCLEIC ACIDS RESEARCH
- C2c2 is a single-component programmable RNA-guided RNA-targeting CRISPR effector
- (2016) Omar O. Abudayyeh et al. SCIENCE
- Structures of human ADAR2 bound to dsRNA reveal base-flipping mechanism and basis for site selectivity
- (2016) Melissa M Matthews et al. NATURE STRUCTURAL & MOLECULAR BIOLOGY
- In vivo Editing of the Human Mutant Rhodopsin Gene by Electroporation of Plasmid-based CRISPR/Cas9 in the Mouse Retina
- (2016) Maria Carmela Latella et al. Molecular Therapy-Nucleic Acids
- A Phenotypic Screen for Functional Mutants of Human Adenosine Deaminase Acting on RNA 1
- (2015) Yuru Wang et al. ACS Chemical Biology
- On the Causes of Evolutionary Transition:Transversion Bias
- (2015) Arlin Stoltzfus et al. MOLECULAR BIOLOGY AND EVOLUTION
- MMEJ-assisted gene knock-in using TALENs and CRISPR-Cas9 with the PITCh systems
- (2015) Tetsushi Sakuma et al. Nature Protocols
- A-to-I editing of coding and non-coding RNAs by ADARs
- (2015) Kazuko Nishikura NATURE REVIEWS MOLECULAR CELL BIOLOGY
- Tissue-based map of the human proteome
- (2015) M. Uhlen et al. SCIENCE
- Improving Site-Directed RNA Editing In Vitro and in Cell Culture by Chemical Modification of the GuideRNA
- (2014) Paul Vogel et al. ANGEWANDTE CHEMIE-INTERNATIONAL EDITION
- CEP290 gene transfer rescues Leber congenital amaurosis cellular phenotype
- (2014) E R Burnight et al. GENE THERAPY
- Down-Regulation of the RNA Editing Enzyme ADAR2 Contributes to RGC Death in a Mouse Model of Glaucoma
- (2014) Ai Ling Wang et al. PLoS One
- Vector platforms for gene therapy of inherited retinopathies
- (2014) Ivana Trapani et al. PROGRESS IN RETINAL AND EYE RESEARCH
- Correction of mutations within the cystic fibrosis transmembrane conductance regulator by site-directed RNA editing
- (2013) M. F. Montiel-Gonzalez et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- An RNA-Deaminase Conjugate Selectively Repairs Point Mutations
- (2012) Thorsten Stafforst et al. ANGEWANDTE CHEMIE-INTERNATIONAL EDITION
- Non-viral retinal gene therapy: a review
- (2011) Peter Charbel Issa et al. CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY
- Increased A-to-I RNA editing of the transcript for GABAA receptor subunit α3 during chick retinal development
- (2010) HENRIK RING et al. VISUAL NEUROSCIENCE
- Identification of a selective nuclear import signal in adenosine deaminases acting on RNA
- (2009) Stefan Maas et al. NUCLEIC ACIDS RESEARCH
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