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

The Lancet (British edition), 2014-03, Vol.383 (9923), p.1129-1137

2014

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Transplanted photoreceptor precursors transfer proteins to host photoreceptors by a mechanism of cytoplasmic fusion

Nature communications, 2016-11, Vol.7 (1), p.13537-13537, Article 13537

2016

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Rescue of the Stargardt phenotype in Abca4 knockout mice through inhibition of vitamin A dimerization

Proceedings of the National Academy of Sciences - PNAS, 2015-07, Vol.112 (27), p.8415-8420

2015

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Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia

Nature medicine, 2018-10, Vol.24 (10), p.1507-1512

2018

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Reversal of end-stage retinal degeneration and restoration of visual function by photoreceptor transplantation

Proceedings of the National Academy of Sciences - PNAS, 2013-01, Vol.110 (3), p.1101-1106

2013

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Visual Acuity after Retinal Gene Therapy for Choroideremia

The New England journal of medicine, 2016-05, Vol.374 (20), p.1996-1998

2016

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Function of human pluripotent stem cell-derived photoreceptor progenitors in blind mice

Scientific reports, 2016-07, Vol.6 (1), p.29784-29784, Article 29784

2016

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RNA editing as a therapeutic approach for retinal gene therapy requiring long coding sequences

International journal of molecular sciences, 2020-01, Vol.21 (3), p.777

2020

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Mirtron-mediated RNA knockdown/replacement therapy for the treatment of dominant retinitis pigmentosa

Nature communications, 2021-08, Vol.12 (1), p.4934-13, Article 4934

2021

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When clocks go bad: neurobehavioural consequences of disrupted circadian timing

PLoS genetics, 2008-05, Vol.4 (5), p.e1000040-e1000040

2008

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Long-term restoration of visual function in end-stage retinal degeneration using subretinal human melanopsin gene therapy

Proceedings of the National Academy of Sciences - PNAS, 2017-10, Vol.114 (42), p.11211-11216

2017

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Immunomodulatory Effects of Hydroxychloroquine and Chloroquine in Viral Infections and Their Potential Application in Retinal Gene Therapy

International journal of molecular sciences, 2020-07, Vol.21 (14), p.4972

2020

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Melanopsin cells are the principal conduits for rod-cone input to non-image-forming vision

Nature, 2008-05, Vol.453 (7191), p.102-105

2008

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The nanophthalmos protein TMEM98 inhibits MYRF self-cleavage and is required for eye size specification

PLoS genetics, 2020-04, Vol.16 (4), p.e1008583-e1008583

2020

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Repair of Retinal Degeneration following Ex Vivo Minicircle DNA Gene Therapy and Transplantation of Corrected Photoreceptor Progenitors

Molecular therapy, 2020-03, Vol.28 (3), p.830-844

2020

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CNTF Gene Therapy Confers Lifelong Neuroprotection in a Mouse Model of Human Retinitis Pigmentosa

Molecular therapy, 2015-08, Vol.23 (8), p.1308-1319

2015

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Filtration of Short-Wavelength Light Provides Therapeutic Benefit in Retinitis Pigmentosa Caused by a Common Rhodopsin Mutation

Investigative ophthalmology & visual science, 2019-06, Vol.60 (7), p.2733-2742

2019

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After-Hours Mutant Reveals a Role for Fbxl3 in Determining Mammalian Circadian Period

Science (American Association for the Advancement of Science), 2007-05, Vol.316 (5826), p.897-900

2007

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Molecular Therapies for Choroideremia

Genes, 2019-09, Vol.10 (10), p.738

2019

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Analysis of Early Cone Dysfunction in an In Vivo Model of Rod-Cone Dystrophy

International journal of molecular sciences, 2020-08, Vol.21 (17), p.6055

2020

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Inclusion of the Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element Enhances AAV2-Driven Transduction of Mouse and Human Retina

Molecular therapy. Nucleic acids, 2017-03, Vol.6, p.198-208

2017

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Assessment of tropism and effectiveness of new primate-derived hybrid recombinant AAV serotypes in the mouse and primate retina

PloS one, 2013-04, Vol.8 (4), p.e60361-e60361

2013

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Absence of Neuroplastin-65 Affects Synaptogenesis in Mouse Inner Hair Cells and Causes Profound Hearing Loss

The Journal of neuroscience, 2016-01, Vol.36 (1), p.222-234

2016

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Early doors (Edo) mutant mouse reveals the importance of period 2 (PER2) PAS domain structure for circadian pacemaking

Proceedings of the National Academy of Sciences - PNAS, 2016-03, Vol.113 (10), p.2756-2761

2016

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Non-image-forming light driven functions are preserved in a mouse model of autosomal dominant optic atrophy

PloS one, 2013-02, Vol.8 (2), p.e56350

2013

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

Transplanted photoreceptor precursors transfer proteins to host photoreceptors by a mechanism of cytoplasmic fusion

Rescue of the Stargardt phenotype in Abca4 knockout mice through inhibition of vitamin A dimerization

Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia

Reversal of end-stage retinal degeneration and restoration of visual function by photoreceptor transplantation

Visual Acuity after Retinal Gene Therapy for Choroideremia

Function of human pluripotent stem cell-derived photoreceptor progenitors in blind mice

RNA editing as a therapeutic approach for retinal gene therapy requiring long coding sequences

Mirtron-mediated RNA knockdown/replacement therapy for the treatment of dominant retinitis pigmentosa

When clocks go bad: neurobehavioural consequences of disrupted circadian timing

Long-term restoration of visual function in end-stage retinal degeneration using subretinal human melanopsin gene therapy

Immunomodulatory Effects of Hydroxychloroquine and Chloroquine in Viral Infections and Their Potential Application in Retinal Gene Therapy

Melanopsin cells are the principal conduits for rod-cone input to non-image-forming vision

The nanophthalmos protein TMEM98 inhibits MYRF self-cleavage and is required for eye size specification

Repair of Retinal Degeneration following Ex Vivo Minicircle DNA Gene Therapy and Transplantation of Corrected Photoreceptor Progenitors

CNTF Gene Therapy Confers Lifelong Neuroprotection in a Mouse Model of Human Retinitis Pigmentosa

Filtration of Short-Wavelength Light Provides Therapeutic Benefit in Retinitis Pigmentosa Caused by a Common Rhodopsin Mutation

After-Hours Mutant Reveals a Role for Fbxl3 in Determining Mammalian Circadian Period

Molecular Therapies for Choroideremia

Analysis of Early Cone Dysfunction in an In Vivo Model of Rod-Cone Dystrophy

Inclusion of the Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element Enhances AAV2-Driven Transduction of Mouse and Human Retina

Assessment of tropism and effectiveness of new primate-derived hybrid recombinant AAV serotypes in the mouse and primate retina

Absence of Neuroplastin-65 Affects Synaptogenesis in Mouse Inner Hair Cells and Causes Profound Hearing Loss

Early doors (Edo) mutant mouse reveals the importance of period 2 (PER2) PAS domain structure for circadian pacemaking

Non-image-forming light driven functions are preserved in a mouse model of autosomal dominant optic atrophy

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