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CRISPR-Cas9 DNA Base-Editing and Prime-Editing

International journal of molecular sciences, 2020-08, Vol.21 (17), p.6240

2020

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Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR

Nature medicine, 2020-03, Vol.26 (3), p.354-359

2020

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Choroideremia Gene Therapy Phase 2 Clinical Trial: 24-Month Results

American journal of ophthalmology, 2019-01, Vol.197, p.65-73

2019

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Two-Year Results After AAV2-Mediated Gene Therapy for Choroideremia: The Alberta Experience

American journal of ophthalmology, 2018-09, Vol.193, p.130-142

2018

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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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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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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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Optogenetic Gene Therapy for the Degenerate Retina: Recent Advances

Frontiers in neuroscience, 2020-11, Vol.14, p.570909-570909

2020

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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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CRISPR Manipulation of Age-Related Macular Degeneration Haplotypes in the Complement System: Potential Future Therapeutic Applications/Avenues

International journal of molecular sciences, 2024-02, Vol.25 (3), p.1697

2024

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Subretinal Visual Implant Alpha IMS – Clinical trial interim report

Vision research (Oxford), 2015-06, Vol.111 (Pt B), p.149-160

2015

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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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Will Nanotechnology Bring New Hope for Gene Delivery?

Trends in biotechnology (Regular ed.), 2017-05, Vol.35 (5), p.434-451

2017

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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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Minicircle Delivery to the Neural Retina as a Gene Therapy Approach

International journal of molecular sciences, 2022-10, Vol.23 (19), p.11673

2022

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Gene Therapy and Stem Cell Transplantation in Retinal Disease: The New Frontier

Ophthalmology (Rochester, Minn.), 2016-10, Vol.123 (10), p.S98-S106

2016

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A Review of CRISPR Tools for Treating Usher Syndrome: Applicability, Safety, Efficiency, and In Vivo Delivery

International journal of molecular sciences, 2023-04, Vol.24 (8), p.7603

2023

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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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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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New CRISPR Tools to Correct Pathogenic Mutations in Usher Syndrome

International journal of molecular sciences, 2022-10, Vol.23 (19), p.11669

2022

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CRISPR Systems Suitable for Single AAV Vector Delivery

Current gene therapy, 2022-01, Vol.22 (1), p.1-14

2022

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Interim Results of a Multicenter Trial with the New Electronic Subretinal Implant Alpha AMS in 15 Patients Blind from Inherited Retinal Degenerations

Frontiers in neuroscience, 2017-08, Vol.11, p.445-445

2017

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RPGR-Related X-Linked Retinitis Pigmentosa Carriers with a Severe “Male Pattern”

Ophthalmologica (Basel), 2021, Vol.244 (1), p.60-67

2021

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Comment on Di Giosaffatte et al. A Novel Hypothesis on Choroideremia-Manifesting Female Carriers: Could CHM In-Frame Variants Exert a Dominant Negative Effect? A Case Report. Genes 2022, 13 , 1268

Genes, 2023-11, Vol.14 (12), p.2160

2023

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CRISPR-Cas9 DNA Base-Editing and Prime-Editing

Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR

Choroideremia Gene Therapy Phase 2 Clinical Trial: 24-Month Results

Two-Year Results After AAV2-Mediated Gene Therapy for Choroideremia: The Alberta Experience

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

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

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

Optogenetic Gene Therapy for the Degenerate Retina: Recent Advances

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

CRISPR Manipulation of Age-Related Macular Degeneration Haplotypes in the Complement System: Potential Future Therapeutic Applications/Avenues

Subretinal Visual Implant Alpha IMS – Clinical trial interim report

Visual Acuity after Retinal Gene Therapy for Choroideremia

Will Nanotechnology Bring New Hope for Gene Delivery?

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

Minicircle Delivery to the Neural Retina as a Gene Therapy Approach

Gene Therapy and Stem Cell Transplantation in Retinal Disease: The New Frontier

A Review of CRISPR Tools for Treating Usher Syndrome: Applicability, Safety, Efficiency, and In Vivo Delivery

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

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

New CRISPR Tools to Correct Pathogenic Mutations in Usher Syndrome

CRISPR Systems Suitable for Single AAV Vector Delivery

Interim Results of a Multicenter Trial with the New Electronic Subretinal Implant Alpha AMS in 15 Patients Blind from Inherited Retinal Degenerations

RPGR-Related X-Linked Retinitis Pigmentosa Carriers with a Severe “Male Pattern”

Comment on Di Giosaffatte et al. A Novel Hypothesis on Choroideremia-Manifesting Female Carriers: Could CHM In-Frame Variants Exert a Dominant Negative Effect? A Case Report. Genes 2022, 13 , 1268

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