A Novel Retinal Ganglion Cell Promoter for Utility in AAV Vectors

Evaluation of Dose and Safety of AAV7m8 and AAV8BP2 in the Non-Human Primate Retina

(2017) Pavitra S. Ramachandran et al.   HUMAN GENE THERAPY

Toward an elucidation of the molecular genetics of inherited retinal degenerations

(2017) G. Jane Farrar et al.   HUMAN MOLECULAR GENETICS

Expression of transcription factors divides retinal ganglion cells into distinct classes

(2017) Neal T. Sweeney et al.   JOURNAL OF COMPARATIVE NEUROLOGY

Single point mutation in adeno-associated viral vectors -DJ capsid leads to improvement for gene delivery in vivo

(2016) Yingying Mao et al.   BMC BIOTECHNOLOGY

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

(2016) Abhishek Sengupta et al.   EMBO Molecular Medicine

Highly Efficient Delivery of Adeno-Associated Viral Vectors to the Primate Retina

(2016) Shannon E. Boye et al.   HUMAN GENE THERAPY

Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial

(2016) Nicola G. Ghazi et al.   HUMAN GENETICS

Amacrine cells coupled to ganglion cells via gap junctions are highly vulnerable in glaucomatous mouse retinas

(2016) Abram Akopian et al.   JOURNAL OF COMPARATIVE NEUROLOGY

Promoter-Specific Effects of DREADD Modulation on Hippocampal Synaptic Plasticity and Memory Formation

(2016) A. J. Lopez et al.   JOURNAL OF NEUROSCIENCE

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

(2016) Jean Bennett et al.   LANCET

The functional diversity of retinal ganglion cells in the mouse

(2016) Tom Baden et al.   NATURE

Neural stem cells from mouse strain Thy1 YFP-16 are a valuable tool to monitor and evaluate neuronal differentiation and morphology

(2016) Ivan Alić et al.   NEUROSCIENCE LETTERS

Gene Therapy for Leber Hereditary Optic Neuropathy

(2016) William J. Feuer et al.   OPHTHALMOLOGY

Two Pairs of ON and OFF Retinal Ganglion Cells Are Defined by Intersectional Patterns of Transcription Factor Expression

(2016) David L. Rousso et al.   Cell Reports

Isolation and Molecular Profiling of Primary Mouse Retinal Ganglion Cells: Comparison of Phenotypes from Healthy and Glaucomatous Retinas

(2016) Sumana R. Chintalapudi et al.   Frontiers in Aging Neuroscience

Vitrectomy Before Intravitreal Injection of AAV2/2 Vector Promotes Efficient Transduction of Retinal Ganglion Cells in Dogs and Nonhuman Primates

(2016) Kizito-Tshitoko Tshilenge et al.   Human Gene Therapy Methods

Novel Methodology for Creating Macaque Retinas with Sortable Photoreceptors and Ganglion Cells

(2016) Shreyasi Choudhury et al.   Frontiers in Neuroscience

Genetic Networks in Mouse Retinal Ganglion Cells

(2016) Felix L. Struebing et al.   Frontiers in Genetics

Long-term outcomes of gene therapy for the treatment of Leber's hereditary optic neuropathy

(2016) Shuo Yang et al.   EBioMedicine

The Types of Retinal Ganglion Cells: Current Status and Implications for Neuronal Classification

(2015) Joshua R. Sanes et al.   Annual Review of Neuroscience

Time-dependent retinal ganglion cell loss, microglial activation and blood-retina-barrier tightness in an acute model of ocular hypertension

(2015) A. Trost et al.   EXPERIMENTAL EYE RESEARCH

Functional Assembly of Accessory Optic System Circuitry Critical for Compensatory Eye Movements

(2015) Lu O. Sun et al.   NEURON

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

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

Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho−/− mouse

(2015) Arpad Palfi et al.   Molecular Therapy-Methods & Clinical Development

AAV-mediated RLBP1 gene therapy improves the rate of dark adaptation in Rlbp1 knockout mice

(2015) Vivian W Choi et al.   Molecular Therapy-Methods & Clinical Development

Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno-associated virus capsid and promoter

(2014) T. Cronin et al.   EMBO Molecular Medicine

Genetically Targeted Binary Labeling of Retinal Neurons

(2014) Y. Zhu et al.   JOURNAL OF NEUROSCIENCE

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial

(2014) Robert E MacLaren et al.   LANCET

Restoration of visual function by expression of a light-gated mammalian ion channel in retinal ganglion cells or ON-bipolar cells

(2014) Benjamin M. Gaub et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Gene therapies for inherited retinal disorders

(2014) G. JANE FARRAR et al.   VISUAL NEUROSCIENCE

Displaced retinal ganglion cells in albino and pigmented rats

(2014) Francisco M. Nadal-Nicolás et al.   Frontiers in Neuroanatomy

Safety and Effects of the Vector for the Leber Hereditary Optic Neuropathy Gene Therapy Clinical Trial

(2014) Rajeshwari D. Koilkonda et al.   JAMA Ophthalmology

Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach

(2013) F M Mowat et al.   GENE THERAPY

RD3 gene delivery restores guanylate cyclase localization and rescues photoreceptors in the Rd3 mouse model of Leber congenital amaurosis 12

(2013) L. L. Molday et al.   HUMAN MOLECULAR GENETICS

Targeting Photoreceptors via Intravitreal Delivery Using Novel, Capsid-Mutated AAV Vectors

(2013) Christine N. Kay et al.   PLoS One

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

Mitochondrial disorders: aetiologies, models systems, and candidate therapies

(2013) G. Jane Farrar et al.   TRENDS IN GENETICS

Intravitreal delivery of AAV-NDI1 provides functional benefit in a murine model of Leber hereditary optic neuropathy

(2012) Naomi Chadderton et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Efficacy of Codelivery of Dual AAV2/5 Vectors in the Murine Retina and Hippocampus

(2012) Arpad Palfi et al.   HUMAN GENE THERAPY

Gene therapy provides long-term visual function in a pre-clinical model of retinitis pigmentosa

(2012) K. J. Wert et al.   HUMAN MOLECULAR GENETICS

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

An optogenetic toolbox designed for primates

(2011) Ilka Diester et al.   NATURE NEUROSCIENCE

Induction of Rapid and Highly Efficient Expression of the Human ND4 Complex I Subunit in the Mouse Visual System by Self-complementary Adeno-Associated Virus

(2010) Rajeshwari D. Koilkonda   ARCHIVES OF OPHTHALMOLOGY

Functional and Behavioral Restoration of Vision by Gene Therapy in the Guanylate Cyclase-1 (GC1) Knockout Mouse

(2010) Shannon E. Boye et al.   PLoS One

Genetic Reactivation of Cone Photoreceptors Restores Visual Responses in Retinitis Pigmentosa

(2010) V. Busskamp et al.   SCIENCE

Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations

(2009) X Sun et al.   GENE THERAPY

Adeno-Associated Virus-Mediated Rhodopsin Replacement Provides Therapeutic Benefit in Mice with a Targeted Disruption of the Rhodopsin Gene

(2009) Arpad Palfi et al.   HUMAN GENE THERAPY

Ectopic Expression of Multiple Microbial Rhodopsins Restores ON and OFF Light Responses in Retinas with Photoreceptor Degeneration

(2009) Y. Zhang et al.   JOURNAL OF NEUROSCIENCE

Clinical gene therapy using recombinant adeno-associated virus vectors

(2008) C Mueller et al.   GENE THERAPY

Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

(2008) William W. Hauswirth et al.   HUMAN GENE THERAPY

Novel AAV serotypes for improved ocular gene transfer

(2008) Corinna Lebherz et al.   JOURNAL OF GENE MEDICINE

High-efficiency Transduction of the Mouse Retina by Tyrosine-mutant AAV Serotype Vectors

(2008) Hilda Petrs-Silva et al.   MOLECULAR THERAPY

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

(2008) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE