Early and late stage gene therapy interventions for inherited retinal degenerations

Host immune responses after suprachoroidal delivery of AAV8 in nonhuman primate eyes.

(2021) Sook Hyun Chung et al.   HUMAN GENE THERAPY

Optogenetic therapy: high spatiotemporal resolution and pattern discrimination compatible with vision restoration in non-human primates

(2021) Gregory Gauvain et al.   Communications Biology

Allele-specific editing ameliorates dominant retinitis pigmentosa in a transgenic mouse model

(2021) Clarissa Patrizi et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses

(2020) Jonathan M. Levy et al.   Nature Biomedical Engineering

Effect of AAV-mediated rhodopsin gene augmentation on retinal degeneration caused by the dominant P23H rhodopsin mutation in a knock-in murine model

(2020) Harry Orlans et al.   HUMAN GENE THERAPY

Microglia modulation by TGF-β1 protects cones in mouse models of retinal degeneration

(2020) Sean K. Wang et al.   JOURNAL OF CLINICAL INVESTIGATION

In vivo–directed evolution of adeno-associated virus in the primate retina

(2020) Leah C. Byrne et al.   JCI Insight

Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing

(2020) Susie Suh et al.   Nature Biomedical Engineering

Restoration of high-sensitivity and adapting vision with a cone opsin

(2019) Michael H. Berry et al.   Nature Communications

AAVcis-regulatory sequences are correlated with ocular toxicity

(2019) Wenjun Xiong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Gene therapy for retinal dystrophy

(2019) José Alain Sahel et al.   NATURE MEDICINE

Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

(2019) Piotr S. Kowalski et al.   MOLECULAR THERAPY

Retinal neuroprotection

(2019) Thomas J. Wubben et al.   CURRENT OPINION IN OPHTHALMOLOGY

Depicting brighter possibilities for treating blindness

(2019) José-Alain Sahel et al.   Science Translational Medicine

Lipid nanoparticles for delivery of messenger RNA to the back of the eye

(2019) Siddharth Patel et al.   JOURNAL OF CONTROLLED RELEASE

Allele-specific gene editing prevents deafness in a model of dominant progressive hearing loss

(2019) Bence György et al.   NATURE MEDICINE

Targeting neuronal and glial cell types with synthetic promoter AAVs in mice, non-human primates and humans

(2019) Josephine Jüttner et al.   NATURE NEUROSCIENCE

Non-viral delivery of chemically modified mRNA to the retina: Subretinal versus intravitreal administration

(2019) Joke Devoldere et al.   JOURNAL OF CONTROLLED RELEASE

Structure comparison of the chimeric AAV2.7m8 vector with parental AAV2

(2019) Antonette Bennett et al.   JOURNAL OF STRUCTURAL BIOLOGY

Genomic form of rhodopsin DNA nanoparticles rescued autosomal dominant Retinitis pigmentosa in the P23H knock-in mouse model

(2018) Rajendra Narayan Mitra et al.   BIOMATERIALS

Gene Therapy for Retinal Degeneration

(2018) Rajendra S. Apte   CELL

Gene therapy and genome surgery in the retina

(2018) James E. DiCarlo et al.   JOURNAL OF CLINICAL INVESTIGATION

In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation

(2018) Ana M. Moreno et al.   MOLECULAR THERAPY

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 comes of age

(2018) Cynthia E. Dunbar et al.   SCIENCE

Special Issue Introduction: Inherited Retinal Disease: Novel Candidate Genes, Genotype–Phenotype Correlations, and Inheritance Models

(2018) Frans Cremers et al.   Genes

Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population

(2018) Vijaya L. Simhadri et al.   Molecular Therapy-Methods & Clinical Development

Delivery approaches for CRISPR/Cas9 therapeutics in vivo: advances and challenges

(2018) D.C. Luther et al.   Expert Opinion on Drug Delivery

Correlation Between Visual Function and Simulated Performance of Daily Living Activities in Glaucomatous Patients

(2018) Marco Lombardi et al.   JOURNAL OF GLAUCOMA

Restoration of vision after de novo genesis of rod photoreceptors in mammalian retinas

(2018) Kai Yao et al.   NATURE

Somatic Gene Editing of GUCY2D by AAV-CRISPR/Cas9 Alters Retinal Structure and Function in Mouse and Macaque

(2018) K. Tyler McCullough et al.   HUMAN GENE THERAPY

Base editing: precision chemistry on the genome and transcriptome of living cells

(2018) Holly A. Rees et al.   NATURE REVIEWS GENETICS

Tropism of engineered and evolved recombinant AAV serotypes in the rd1 mouse and ex vivo primate retina

(2017) D G Hickey et al.   GENE THERAPY

Genome surgery using Cas9 ribonucleoproteins for the treatment of age-related macular degeneration

(2017) Kyoungmi Kim et al.   GENOME RESEARCH

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

Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward

(2017) Jean Bennett   MOLECULAR THERAPY

Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents

(2017) Xue Gao et al.   NATURE

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

(2017) Nicole M. Gaudelli 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

Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice

(2017) Wenhan Yu et al.   Nature Communications

The Thioredoxin Encoded by the Rod-Derived Cone Viability Factor Gene Protects Cone Photoreceptors Against Oxidative Stress

(2016) Xin Mei et al.   ANTIOXIDANTS & REDOX SIGNALING

Structural recovery of the retina in a retinoschisin-deficient mouse after gene replacement therapy by solid lipid nanoparticles

(2016) P.S. Apaolaza et al.   BIOMATERIALS

Red‐shifted channelrhodopsin stimulation restores light responses in blind mice, macaque retina, and human retina

(2016) Abhishek Sengupta et al.   EMBO Molecular Medicine

Application of lipid nanoparticles to ocular drug delivery

(2016) Luigi Battaglia et al.   Expert Opinion on Drug Delivery

Let There Be Light: Gene and Cell Therapy for Blindness

(2016) Deniz Dalkara et al.   HUMAN GENE THERAPY

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

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor et al.   NATURE

Five-Year Safety and Performance Results from the Argus II Retinal Prosthesis System Clinical Trial

(2016) Lyndon da Cruz et al.   OPHTHALMOLOGY

Retinal stimulation strategies to restore vision: Fundamentals and systems

(2016) Lan Yue et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Emerging therapies for inherited retinal degeneration

(2016) H. P. N. Scholl et al.   Science Translational Medicine

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

Gene therapy in LHON

(2015) C. Vignal-Clermont et al.   ACTA OPHTHALMOLOGICA

Rod-Derived Cone Viability Factor Promotes Cone Survival by Stimulating Aerobic Glycolysis

(2015) Najate Aït-Ali et al.   CELL

Small Molecules Enhance CRISPR Genome Editing in Pluripotent Stem Cells

(2015) Chen Yu et al.   Cell Stem Cell

Nanoparticle-based technologies for retinal gene therapy

(2015) Jeffrey Adijanto et al.   EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS

Adeno-Associated Virus at 50: A Golden Anniversary of Discovery, Research, and Gene Therapy Success—A Personal Perspective

(2015) Eric Hastie et al.   HUMAN GENE THERAPY

Exploiting metabolic and antioxidant pathways to maintain vision in blinding disease

(2015) Pavitra S. Ramachandran et al.   JOURNAL OF CLINICAL INVESTIGATION

Activated mTORC1 promotes long-term cone survival in retinitis pigmentosa mice

(2015) Aditya Venkatesh et al.   JOURNAL OF CLINICAL INVESTIGATION

NRF2 promotes neuronal survival in neurodegeneration and acute nerve damage

(2015) Wenjun Xiong et al.   JOURNAL OF CLINICAL INVESTIGATION

Optogenetic Vision Restoration Using Rhodopsin for Enhanced Sensitivity

(2015) Benjamin M Gaub et al.   MOLECULAR THERAPY

Targeting Channelrhodopsin-2 to ON-bipolar Cells With Vitreally Administered AAV Restores ON and OFF Visual Responses in Blind Mice

(2015) Emilie Macé et al.   MOLECULAR THERAPY

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining

(2015) Takeshi Maruyama et al.   NATURE BIOTECHNOLOGY

Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

(2015) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

Improvement and Decline in Vision with Gene Therapy in Childhood Blindness

(2015) Samuel G. Jacobson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Congenital stationary night blindness: An analysis and update of genotype–phenotype correlations and pathogenic mechanisms

(2015) Christina Zeitz et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Rationally engineered Cas9 nucleases with improved specificity

(2015) I. M. Slaymaker et al.   SCIENCE

Restoring the ON Switch in Blind Retinas: Opto-mGluR6, a Next-Generation, Cell-Tailored Optogenetic Tool

(2015) Michiel van Wyk et al.   PLOS BIOLOGY

Investor Outlook: Focus on Upcoming LCA2 Gene Therapy Phase III Results

(2015) Joshua Schimmer et al.   Human Gene Therapy Clinical Development

Retinal transduction profiles by high-capacity viral vectors

(2014) A Puppo et al.   GENE THERAPY

Viral-mediated RdCVF and RdCVFL expression protects cone and rod photoreceptors in retinal degeneration

(2014) Leah C. Byrne et al.   JOURNAL OF CLINICAL INVESTIGATION

Restoration of the Majority of the Visual Spectrum by Using Modified Volvox Channelrhodopsin-1

(2014) Hiroshi Tomita et al.   MOLECULAR THERAPY

Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo

(2014) John A Zuris et al.   NATURE BIOTECHNOLOGY

Noninvasive optical inhibition with a red-shifted microbial rhodopsin

(2014) Amy S Chuong et al.   NATURE NEUROSCIENCE

Engineering adeno-associated viruses for clinical gene therapy

(2014) Melissa A. Kotterman et al.   NATURE REVIEWS GENETICS

Microglial NADPH oxidase activation mediates rod cell death in the retinal degeneration in rd mice

(2014) H. Zeng et al.   NEUROSCIENCE

Vector platforms for gene therapy of inherited retinopathies

(2014) Ivana Trapani et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Trophic factors in the pathogenesis and therapy for retinal degenerative diseases

(2014) Anton M. Kolomeyer et al.   SURVEY OF OPHTHALMOLOGY

Gene Augmentation for adRP Mutations in RHO

(2014) A. S. Lewin et al.   Cold Spring Harbor Perspectives in Medicine

Gene Therapy for Blindness

(2013) José-Alain Sahel et al.   Annual Review of Neuroscience

Synthesis and Characterization of Glycol Chitosan DNA Nanoparticles for Retinal Gene Delivery

(2013) Rajendra N. Mitra et al.   ChemMedChem

Genes and mutations causing retinitis pigmentosa

(2013) S P Daiger et al.   CLINICAL GENETICS

Functional rescue of cone photoreceptors in retinitis pigmentosa

(2013) José-Alain Sahel et al.   GRAEFES ARCHIVE FOR CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY

Receptor Interacting Protein Kinase-Mediated Necrosis Contributes to Cone and Rod Photoreceptor Degeneration in the Retina Lacking Interphotoreceptor Retinoid-Binding Protein

(2013) K. Sato et al.   JOURNAL OF NEUROSCIENCE

Use of lentiviral vectors to deliver and express bicistronic transgenes in developing chicken embryos

(2013) Susan L. Semple-Rowland et al.   METHODS

Seven new loci associated with age-related macular degeneration

(2013) Lars G Fritsche et al.   NATURE GENETICS

ReaChR: a red-shifted variant of channelrhodopsin enables deep transcranial optogenetic excitation

(2013) John Y Lin et al.   NATURE NEUROSCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

In Vivo-Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous

(2013) D. Dalkara et al.   Science Translational Medicine

Oligochitosan polyplexes as carriers for retinal gene delivery

(2012) G. Puras et al.   EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES

Long-Term Rescue of Retinal Structure and Function by Rhodopsin RNA Replacement with a Single Adeno-Associated Viral Vector in P23H RHO Transgenic Mice

(2012) Haoyu Mao et al.   HUMAN GENE THERAPY

DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice

(2012) Zongchao Han et al.   JOURNAL OF CLINICAL INVESTIGATION

Comparative Analysis of DNA Nanoparticles and AAVs for Ocular Gene Delivery

(2012) Zongchao Han et al.   PLoS One

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa

(2011) Claudio Mussolino et al.   EMBO Molecular Medicine

Imaging retinal mosaics in the living eye

(2011) E A Rossi et al.   EYE

Gene-based therapies for dominantly inherited retinopathies

(2011) G J Farrar et al.   GENE THERAPY

Gene Transfer for Neovascular Age-Related Macular Degeneration

(2011) Peter A. Campochiaro   HUMAN GENE THERAPY

Suppression and Replacement Gene Therapy for Autosomal Dominant Disease in a Murine Model of Dominant Retinitis Pigmentosa

(2011) Sophia Millington-Ward et al.   MOLECULAR THERAPY

AAV Mediated GDNF Secretion From Retinal Glia Slows Down Retinal Degeneration in a Rat Model of Retinitis Pigmentosa

(2011) Deniz Dalkara et al.   MOLECULAR THERAPY

Melanopsin signalling in mammalian iris and retina

(2011) T. Xue et al.   NATURE

Bicistronic Lentiviruses Containing a Viral 2A Cleavage Sequence Reliably Co-Express Two Proteins and Restore Vision to an Animal Model of LCA1

(2011) Jonathan D. Verrier et al.   PLoS One

Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model

(2011) L. Jiang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Gene Therapy in Ophthalmology: Validation on Cultured Retinal Cells and Explants from Postmortem Human Eyes

(2010) Mathias Fradot et al.   HUMAN GENE THERAPY

AAV Delivery of Wild-Type Rhodopsin Preserves Retinal Function in a Mouse Model of Autosomal Dominant Retinitis Pigmentosa

(2010) Haoyu Mao et al.   HUMAN GENE THERAPY

20 years of gene therapy for SCID

(2010) Alain Fischer et al.   NATURE IMMUNOLOGY

Photoreceptor degeneration: genetic and mechanistic dissection of a complex trait

(2010) Alan F. Wright et al.   NATURE REVIEWS GENETICS

Genetic Reactivation of Cone Photoreceptors Restores Visual Responses in Retinitis Pigmentosa

(2010) V. Busskamp et al.   SCIENCE

Rod-Derived Cone Viability Factor for Treating Blinding Diseases: From Clinic to Redox Signaling

(2010) T. Leveillard et al.   Science Translational Medicine

Mechanisms of Apoptosis in Retinitis Pigmentosa

(2009) Sandra Cottet et al.   CURRENT MOLECULAR MEDICINE

Progress toward effective treatments for human photoreceptor degenerations

(2009) Edwin M Stone   CURRENT OPINION IN GENETICS & DEVELOPMENT

Equine Infectious Anemia Viral Vector-Mediated Codelivery of Endostatin and Angiostatin Driven by Retinal Pigmented Epithelium-Specific VMD2 Promoter Inhibits Choroidal Neovascularization

(2009) Shu Kachi et al.   HUMAN GENE THERAPY

Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice

(2009) Susie E. Barker et al.   JOURNAL OF GENE MEDICINE

Success in sight: The eyes have it! Ocular gene therapy trials for LCA look promising

(2008) J W Bainbridge et al.   GENE THERAPY

Stimulation of the insulin/mTOR pathway delays cone death in a mouse model of retinitis pigmentosa

(2008) Claudio Punzo et al.   NATURE NEUROSCIENCE

Red-shifted optogenetic excitation: a tool for fast neural control derived from Volvox carteri

(2008) Feng Zhang et al.   NATURE NEUROSCIENCE

Restoration of visual function in retinal degeneration mice by ectopic expression of melanopsin

(2008) B. Lin et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Leber congenital amaurosis: Genes, proteins and disease mechanisms

(2008) Anneke I. den Hollander et al.   PROGRESS IN RETINAL AND EYE RESEARCH