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Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery

Molecular therapy, 2018-09, Vol.26 (9), p.2282-2294

2018

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Ocular localization and transduction by adenoviral vectors are serotype-dependent and can be modified by inclusion of RGD fiber modifications

PloS one, 2014-09, Vol.9 (9), p.e108071-e108071

2014

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“Para-retinal” Vector Administration into the Deep Vitreous Enhances Retinal Transgene Expression

Molecular therapy. Methods & clinical development, 2020-09, Vol.18, p.422-427

2020

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Ocular and systemic safety of a recombinant AAV8 vector for X-linked retinoschisis gene therapy: GLP studies in rabbits and Rs1-KO mice

Molecular therapy. Methods & clinical development, 2016-01, Vol.5 (C), p.16011-16011, Article 16011

2016

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Immune function in X-linked retinoschisis subjects in an AAV8-RS1 phase I/IIa gene therapy trial

Molecular therapy, 2021-06, Vol.29 (6), p.2030-2040

2021

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Retinal Neuroprotection against Ischemic Injury Mediated by Intraocular Gene Transfer of Pigment Epithelium-Derived Factor

Investigative ophthalmology & visual science, 2003-10, Vol.44 (10), p.4497-4504

2003

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Intraocular gene transfer of pigment epithelium-derived factor rescues photoreceptors from light-induced cell death

Journal of cellular physiology, 2005-02, Vol.202 (2), p.570-578

2005

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Adenoviral vector-delivered pigment epithelium-derived factor for neovascular age-related macular degeneration: results of a phase I clinical trial

Human gene therapy, 2006-02, Vol.17 (2), p.167

2006

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Intravitreal Ciliary Neurotrophic Factor Transiently Improves Cone-Mediated Function in a CNGB3-/- Mouse Model of Achromatopsia

Investigative ophthalmology & visual science, 2015-10, Vol.56 (11), p.6810-6822

2015

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CNGB3-achromatopsia clinical trial with CNTF: diminished rod pathway responses with no evidence of improvement in cone function

Investigative ophthalmology & visual science, 2014-10, Vol.55 (10), p.6301-6308

2014

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Convergence of Human Genetics and Animal Studies: Gene Therapy for X-Linked Retinoschisis

Cold Spring Harbor perspectives in medicine, 2015-08, Vol.5 (8), p.a017368-a017368

2015

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Pigment epithelium-derived factor inhibits retinal and choroidal neovascularization

Journal of cellular physiology, 2001-08, Vol.188 (2), p.253-263

2001

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Preclinical safety evaluation of a recombinant AAV8 vector for X-linked retinoschisis after intravitreal administration in rabbits

Human gene therapy. Clinical development, 2014-12, Vol.25 (4), p.202

2014

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Overexpression of KAI1 suppresses in vitro invasiveness and in vivo metastasis in breast cancer cells

Cancer research (Chicago, Ill.), 2001-07, Vol.61 (13), p.5284-5288

2001

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Alternate serotype adenovector provides long-term therapeutic gene expression in the eye

Molecular vision, 2008-12, Vol.14, p.2535-2546

2008

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Instrument-based vision screening: update and review.(peds v2.0)

Contemporary pediatrics (Montvale, N.J.), 2014-02, Vol.31 (2), p.39

2014

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The role of lacrimal intubation in the management of facial trauma and tumor resection

Plastic and reconstructive surgery (1963), 2005-06, Vol.115 (7), p.1871-1876

2005

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Repeat Administration of Proteins to the Eye With a Single Intraocular Injection of an Adenovirus Vector

Molecular therapy, 2008-08, Vol.16 (8), p.1444-1449

2008

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Trans-scleral delivery of antiangiogenic proteins

Journal of ocular pharmacology and therapeutics, 2008-02, Vol.24 (1), p.70

2008

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Persistent Expression of PEDF in the Eye Using High-capacity Adenovectors

Molecular therapy, 2008-12, Vol.16 (12), p.1986-1994

2008

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Repeated Administration of Adenovector in the Eye Results in Efficient Gene Delivery

Investigative ophthalmology & visual science, 2006-01, Vol.47 (1), p.299-305

2006

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A unilateral white pupil in a 6-year-old girl.(PUZZLER)(Case study)

Contemporary pediatrics (Montvale, N.J.), 2013-02, Vol.30 (2), p.34

2013

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Laser Photocoagulation and, to a Lesser Extent, Photodynamic Therapy Target and Enhance Adenovirus Vector-Mediated Gene Transfer in the Rat Retina

Investigative ophthalmology & visual science, 2005-10, Vol.46 (10), p.3883-3891

2005

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Evidence for the existence of a third progesterone receptor protein in human breast cancer cell line T47D

Cancer research (Chicago, Ill.), 1994-01, Vol.54 (2), p.340-343

1994

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Suppression of MDA-MB-435 breast carcinoma cell metastasis following the introduction of human chromosome 11

Cancer research (Chicago, Ill.), 1996-03, Vol.56 (6), p.1222-1227

1996

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Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery

Ocular localization and transduction by adenoviral vectors are serotype-dependent and can be modified by inclusion of RGD fiber modifications

“Para-retinal” Vector Administration into the Deep Vitreous Enhances Retinal Transgene Expression

Ocular and systemic safety of a recombinant AAV8 vector for X-linked retinoschisis gene therapy: GLP studies in rabbits and Rs1-KO mice

Immune function in X-linked retinoschisis subjects in an AAV8-RS1 phase I/IIa gene therapy trial

Retinal Neuroprotection against Ischemic Injury Mediated by Intraocular Gene Transfer of Pigment Epithelium-Derived Factor

Intraocular gene transfer of pigment epithelium-derived factor rescues photoreceptors from light-induced cell death

Adenoviral vector-delivered pigment epithelium-derived factor for neovascular age-related macular degeneration: results of a phase I clinical trial

Intravitreal Ciliary Neurotrophic Factor Transiently Improves Cone-Mediated Function in a CNGB3-/- Mouse Model of Achromatopsia

CNGB3-achromatopsia clinical trial with CNTF: diminished rod pathway responses with no evidence of improvement in cone function

Convergence of Human Genetics and Animal Studies: Gene Therapy for X-Linked Retinoschisis

Pigment epithelium-derived factor inhibits retinal and choroidal neovascularization

Preclinical safety evaluation of a recombinant AAV8 vector for X-linked retinoschisis after intravitreal administration in rabbits

Overexpression of KAI1 suppresses in vitro invasiveness and in vivo metastasis in breast cancer cells

Alternate serotype adenovector provides long-term therapeutic gene expression in the eye

Instrument-based vision screening: update and review.(peds v2.0)

The role of lacrimal intubation in the management of facial trauma and tumor resection

Repeat Administration of Proteins to the Eye With a Single Intraocular Injection of an Adenovirus Vector

Trans-scleral delivery of antiangiogenic proteins

Persistent Expression of PEDF in the Eye Using High-capacity Adenovectors

Repeated Administration of Adenovector in the Eye Results in Efficient Gene Delivery

A unilateral white pupil in a 6-year-old girl.(PUZZLER)(Case study)

Laser Photocoagulation and, to a Lesser Extent, Photodynamic Therapy Target and Enhance Adenovirus Vector-Mediated Gene Transfer in the Rat Retina

Evidence for the existence of a third progesterone receptor protein in human breast cancer cell line T47D

Suppression of MDA-MB-435 breast carcinoma cell metastasis following the introduction of human chromosome 11

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