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Title
Revolution in Gene Medicine Therapy and Genome Surgery
Authors
Keywords
-
Journal
Genes
Volume 9, Issue 12, Pages 575
Publisher
MDPI AG
Online
2018-11-27
DOI
10.3390/genes9120575
References
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Note: Only part of the references are listed.- Gene therapy and genome surgery in the retina
- (2018) James E. DiCarlo et al. JOURNAL OF CLINICAL INVESTIGATION
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- (2018) Andrea Maddalena et al. MOLECULAR THERAPY
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- (2018) Lyndon da Cruz et al. NATURE BIOTECHNOLOGY
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- (2018) Yi-Ting Tsai et al. OPHTHALMOLOGY
- Rewritable multi-event analog recording in bacterial and mammalian cells
- (2018) Weixin Tang et al. SCIENCE
- A bioengineered retinal pigment epithelial monolayer for advanced, dry age-related macular degeneration
- (2018) Amir H. Kashani et al. Science Translational Medicine
- CRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration
- (2018) Taeyoung Koo et al. Nature Communications
- Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models
- (2018) Kalyan Dulla et al. Molecular Therapy-Nucleic Acids
- Lentiviral Vector Gene Transfer of Endostatin/Angiostatin for Macular Degeneration (GEM) Study
- (2017) Peter A. Campochiaro et al. HUMAN GENE THERAPY
- Infection-related microcephaly after the 2015 and 2016 Zika virus outbreaks in Brazil: a surveillance-based analysis
- (2017) Wanderson Kleber de Oliveira et al. LANCET
- CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10
- (2017) Guo-Xiang Ruan et al. MOLECULAR THERAPY
- Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage
- (2017) Nicole M. Gaudelli et al. NATURE
- Vision Loss after Intravitreal Injection of Autologous “Stem Cells” for AMD
- (2017) Ajay E. Kuriyan et al. NEW ENGLAND JOURNAL OF MEDICINE
- Autologous Induced Stem-Cell–Derived Retinal Cells for Macular Degeneration
- (2017) Michiko Mandai et al. NEW ENGLAND JOURNAL OF MEDICINE
- CRISPR/Cpf1 enables fast and simple genome editing of Saccharomyces cerevisiae
- (2017) René Verwaal et al. YEAST
- In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni
- (2017) Eunji Kim et al. Nature Communications
- CRISPR-Cpf1 mediates efficient homology-directed repair and temperature-controlled genome editing
- (2017) Miguel A. Moreno-Mateos et al. Nature Communications
- Applications of CRISPR/Cas9 in retinal degenerative diseases
- (2017) International Journal of Ophthalmology
- Microhomology-mediated end joining: new players join the team
- (2017) Hailong Wang et al. Cell and Bioscience
- Retinal Gene Therapy: Surgical Vector Delivery in the Translation to Clinical Trials
- (2017) G. Alex Ochakovski et al. Frontiers in Neuroscience
- Correction of Monogenic and Common Retinal Disorders with Gene Therapy
- (2017) Jesse Sengillo et al. Genes
- Stargardt disease: clinical features, molecular genetics, animal models and therapeutic options
- (2016) Preena Tanna et al. BRITISH JOURNAL OF OPHTHALMOLOGY
- In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration
- (2016) Keiichiro Suzuki et al. NATURE
- Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
- (2016) Alexis C. Komor et al. NATURE
- High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects
- (2016) Benjamin P. Kleinstiver et al. NATURE
- Multiplex gene editing by CRISPR–Cpf1 using a single crRNA array
- (2016) Bernd Zetsche et al. NATURE BIOTECHNOLOGY
- Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood–Onset Retinal Dystrophy
- (2016) Richard G. Weleber et al. OPHTHALMOLOGY
- Gene Therapy for Leber Hereditary Optic Neuropathy
- (2016) William J. Feuer et al. OPHTHALMOLOGY
- Rhodopsin targeted transcriptional silencing by DNA-binding
- (2016) Salvatore Botta et al. eLife
- 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
- 55. Preclinical Safety Studies for AAV2-MerTK Gene Therapy Vector for Retinitis Pigmentosa
- (2016) MOLECULAR THERAPY
- Precision Medicine: Genetic Repair of Retinitis Pigmentosa in Patient-Derived Stem Cells
- (2016) Alexander G. Bassuk et al. Scientific Reports
- Multimodal Imaging of Central Retinal Disease Progression in a 2-Year Mean Follow-up of Retinitis Pigmentosa
- (2015) Tharikarn Sujirakul et al. AMERICAN JOURNAL OF OPHTHALMOLOGY
- Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System
- (2015) Bernd Zetsche et al. CELL
- Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease
- (2015) Ivana Trapani et al. HUMAN MOLECULAR GENETICS
- Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies
- (2015) Steven D Schwartz et al. LANCET
- T cells expressing CD19 chimeric antigen receptors for acute lymphoblastic leukaemia in children and young adults: a phase 1 dose-escalation trial
- (2015) Daniel W Lee et al. LANCET
- 687. Therapeutic Correction of an LCA-Causing Splice Defect in the CEP290 Gene by CRISPR/Cas-Mediated Genome Editing
- (2015) Morgan L. Maeder et al. MOLECULAR THERAPY
- Retinal transduction profiles by high-capacity viral vectors
- (2014) A Puppo et al. GENE THERAPY
- Intravitreal Autologous Bone Marrow CD34+ Cell Therapy for Ischemic and Degenerative Retinal Disorders: Preliminary Phase 1 Clinical Trial Findings
- (2014) S. S. Park et al. INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE
- Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial
- (2014) Robert E MacLaren et al. LANCET
- EIAV-Based Retinal Gene Therapy in the shaker1 Mouse Model for Usher Syndrome Type 1B: Development of UshStat
- (2014) Marisa Zallocchi et al. PLoS One
- Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression
- (2013) Lei S. Qi et al. CELL
- Effective delivery of large genes to the retina by dual AAV vectors
- (2013) Ivana Trapani et al. EMBO Molecular Medicine
- Unknown
- (2013) Yao Li et al. MOLECULAR MEDICINE
- High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells
- (2013) Yanfang Fu et al. NATURE BIOTECHNOLOGY
- Three-Year Follow-up after Unilateral Subretinal Delivery of Adeno-Associated Virus in Patients with Leber Congenital Amaurosis Type 2
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- (2012) W. A. Beltran et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
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- AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness
- (2012) J. Bennett et al. Science Translational Medicine
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- Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa
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- Directed Differentiation of Human Embryonic Stem Cells into Functional Retinal Pigment Epithelium Cells
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- (2009) Amanda-Jayne Carr et al. PLoS One
- Long-Term Safety and Function of RPE from Human Embryonic Stem Cells in Preclinical Models of Macular Degeneration
- (2009) Bin Lu et al. STEM CELLS
- Elucidating the phenomenon of HESC-derived RPE: Anatomy of cell genesis, expansion and retinal transplantation
- (2008) Anthony Vugler et al. EXPERIMENTAL NEUROLOGY
- Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy
- (2008) J Kong et al. GENE THERAPY
- Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice
- (2008) Mariacarmela Allocca et al. JOURNAL OF CLINICAL INVESTIGATION
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