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ARVO Annual Meeting Abstract June 2020

Gene augmentation therapy in a large animal model of PDE6A -Retinitis Pigmentosa results in functional and structural rescue for at least 2.5 years.

Laurence Mireille Occelli; Stylianos Michalakis; Martin Biel; consortium RD-Cure; Simon M Petersen-Jones

Invest. Ophthalmol. Vis. Sci.. 2020; 61(7):1913. doi:

ARVO Annual Meeting Abstract June 2020

Testing of a therapeutic vector for CNGB1 retinitis pigmentosa

Simon M Petersen-Jones; Laurence Mireille Occelli; Johanna Wagner; Luis Marinho; Amy Frederick; Catherine O'Riordan; Stylianos Michalakis

Invest. Ophthalmol. Vis. Sci.. 2020; 61(7):793. doi:

ARVO Annual Meeting Abstract July 2019

The Contribution of Endoplasmic Reticulum Calcium Deficiency to Mistrafficking of Cone Outer Segment Proteins in CNG Channel Deficiency

Hongwei Ma; Fan Yang; Michael Robert Butler; Jacob Rapp; Yun-Zheng Le; Katsuhiko Mikoshiba; Martin Biel; Stylianos Michalakis; Xi-Qin Ding

Invest. Ophthalmol. Vis. Sci.. 2019; 60(9):4207. doi:

ARVO Annual Meeting Abstract July 2019

The Role of Endoplasmic Reticulum (ER) Calcium Channels in ER Stress and Cone Death in CNG Channel Deficiency

Michael Robert Butler; Hongwei Ma; Fan Yang; Jacob Rapp; Yun-Zheng Le; Katsuhiko Mikoshiba; Martin Biel; Stylianos Michalakis; Xi-Qin Ding

Invest. Ophthalmol. Vis. Sci.. 2019; 60(9):4864. doi:

ARVO Annual Meeting Abstract July 2019

Digenic triallelic inheritance in cone photoreceptor cyclic nucleotide gated channel associated retinopathies

Susanne Kohl; Markus Burkard; Timm Kraetzig; Naoyuki Tanimoto; Britta Baumann; Martin Biel; Robert Lukowski; Mathias W Seeliger; Stylianos Michalakis; Bernd Wissinger; Peter Ruth

Invest. Ophthalmol. Vis. Sci.. 2019; 60(9):419. doi:

ARVO Annual Meeting Abstract July 2019

Effects of Subretinal AAV8 Gene Therapy on Microperimetry in CNGA3 Achromatopsia Patients

G. Alex Ochakovski; Ahmad Zhour; Laura Kuehlewein; Susanne Kohl; Andrea Rindtorff; Karl Ulrich Bartz-Schmidt; Marius Ueffing; Eberhart Zrenner; Stylianos Michalakis; Martin Biel; Bernd Wissinger; Tobias Peters; Barbara Wilhelm; M. Dominik Fischer

Invest. Ophthalmol. Vis. Sci.. 2019; 60(9):2921. doi:

ARVO Annual Meeting Abstract July 2018

Evaluation of reconstitution efficiencies of rAAV-dCas9-VPR split-intein protein trans-splicing vectors in retinal cells

Victoria Splith; Sybille Boehm; Johanna Wagner; Martin Biel; Elvir Becirovic; Stylianos Michalakis

Invest. Ophthalmol. Vis. Sci.. 2018; 59(9):5663. doi:

ARVO Annual Meeting Abstract July 2018

Preclinical evaluation of rAAV.CNGB1 in the Cngb1 knockout mouse model of retinitis pigmentosa.

Johanna Wagner; Christian Schön; Catherine O'Riordan; Abraham Scaria; Seng Cheng; Martin Biel; Stylianos Michalakis

Invest. Ophthalmol. Vis. Sci.. 2018; 59(9):5664. doi:

ARVO Annual Meeting Abstract July 2018

First Gene Supplementation Therapy for CNGA3-linked Achromatopsia

M Dominik Fischer; Stylianos Michalakis; Barbara Wilhelm; Nadine Kahle; Tobias Peters; Karl Ulrich Bartz-Schmidt; Susanne Kohl; Mathias W Seeliger; Nicole Weisschuh; Ditta Zobor; Eberhart Zrenner; Marius Ueffing; Martin Biel; Bernd Wissinger

Invest. Ophthalmol. Vis. Sci.. 2018; 59(9):2982. doi:

ARVO Annual Meeting Abstract July 2018

cGMP/PKG Signaling Regulation of Endoplasmic Reticulum Calcium Channels in Cyclic Nucleotide-gated Channel-deficient Cones

Fan Yang; Hongwei Ma; Michael Butler; Wolfgang Baehr; Martin Biel; Stylianos Michalakis; Xi-Qin Ding

Invest. Ophthalmol. Vis. Sci.. 2018; 59(9):4966. doi:

ARVO Annual Meeting Abstract July 2018

New insights into the mechanisms of retinal degeneration due to Phosphodiesterase 6 (PDE6) deficiency

Regine Lotte Muehlfriedel; Vithiyanjali Sothilingam; Marina Garcia Garrido; Naoyuki Tanimoto; Susanne Beck; Stylianos Michalakis; Martin Biel; Francois Paquet-Durand; Mathias W Seeliger

Invest. Ophthalmol. Vis. Sci.. 2018; 59(9):998. doi:

ARVO Annual Meeting Abstract July 2018

Safety study in cynomolgus monkeys after subretinal injection of rAAV.hPDE6A

Tobias Peters; Barbara Wilhelm; Birgit Korbmacher; Sven Korte; Karl Ulrich Bartz-Schmidt; Stylianos Michalakis; Guy Alex Ochakovski; M Dominik Fischer

Invest. Ophthalmol. Vis. Sci.. 2018; 59(9):2658. doi:

Retina April 2018

Humoral Immune Response After Intravitreal But Not After Subretinal AAV8 in Primates and Patients

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Felix F. Reichel; Tobias Peters; Barbara Wilhelm; Martin Biel; Marius Ueffing; Bernd Wissinger; Karl U. Bartz-Schmidt; Reinhild Klein; Stylianos Michalakis; M. Dominik Fischer; for the RD-CURE Consortium

Invest. Ophthalmol. Vis. Sci.. 2018; 59(5):1910-1915. doi: https://doi.org/10.1167/iovs.17-22494

Includes: Supplemental Content

Retina November 2017

Superior Retinal Gene Transfer and Biodistribution Profile of Subretinal Versus Intravitreal Delivery of AAV8 in Nonhuman Primates

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Immanuel P. Seitz; Stylianos Michalakis; Barbara Wilhelm; Felix F. Reichel; G. Alex Ochakovski; Eberhart Zrenner; Marius Ueffing; Martin Biel; Bernd Wissinger; Karl U. Bartz-Schmidt; Tobias Peters; M. Dominik Fischer; for the RD-CURE Consortium
TAGS: cohort studies, disease vectors, dna, gene transfer techniques, genome, injections, primates, spatial vectors, body tissue, surgical procedures, operative
Invest. Ophthalmol. Vis. Sci.. 2017; 58(13):5792-5801. doi: https://doi.org/10.1167/iovs.17-22473

Includes: Supplemental Content

Retina August 2017

Subretinal Injection for Gene Therapy Does Not Cause Clinically Significant Outer Nuclear Layer Thinning in Normal Primate Foveae

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G. Alex Ochakovski; Tobias Peters; Stylianos Michalakis; Barbara Wilhelm; Bernd Wissinger; Martin Biel; K. Ulrich Bartz-Schmidt; M. Dominik Fischer; for the RD-CURE Consortium
TAGS: gene therapy, injections, primates, safety, serotonin uptake inhibitors, retina, eye, surgical procedures, operative
Invest. Ophthalmol. Vis. Sci.. 2017; 58(10):4155-4160. doi: https://doi.org/10.1167/iovs.17-22402

Includes: Supplemental Content

ARVO Annual Meeting Abstract June 2017

Peripherin-2 and Rom-1 Have Opposing Effects on Rod Outer Segment Targeting of Two Misfolded Peripherin-2 Mutants in Rods

Elvir Becirovic; Sybille Böhm; Lisa Maria Riedmayr; Andreas Gießl; Christian Schön; Stylianos Michalakis; Martin Biel

Invest. Ophthalmol. Vis. Sci.. 2017; 58(8):3564. doi:

ARVO Annual Meeting Abstract June 2017

Evaluation of electroretinography in cynomolgus monkeys after subretinal injection of rAAV.hPDE6A

Tobias Peters; Barbara Wilhelm; birgit niggemann; Sven Korte; Karl Ulrich Bartz-Schmidt; Stylianos Michalakis; Guy Alex Ochakovski; Dominik M Fischer

Invest. Ophthalmol. Vis. Sci.. 2017; 58(8):4096. doi:

ARVO Annual Meeting Abstract June 2017

Non-inferiority of subretinal versus intravitreal injection regarding photoreceptor layer thickness

Guy Alex Ochakovski; Tobias Peters; Stylianos Michalakis; Barbara Wilhelm; K. Ulrich Bartz-Schmidt; M Dominik Fischer

Invest. Ophthalmol. Vis. Sci.. 2017; 58(8):4100. doi:

ARVO Annual Meeting Abstract June 2017

Preclinical evaluation of rAAV8.CNGA3 in the Cnga3 knockout mouse model of ACHM2

Stylianos Michalakis; Christian Schön; Regine Mühlfriedel; Vithiyanjali Sothilingam; Bernd Wissinger; Mathias W Seeliger; Martin Biel

Invest. Ophthalmol. Vis. Sci.. 2017; 58(8):4499. doi:

ARVO Annual Meeting Abstract June 2017

Preclinical evaluation of rAAV8.PDE6A in the Pde6a D670G mutant mouse model of RP43

Christian Schön; Regine Mühlfriedel; Vithiyanjali Sothilingam; Francois Paquet-Durand; Bernd Wissinger; Mathias W Seeliger; Martin Biel; Stylianos Michalakis

Invest. Ophthalmol. Vis. Sci.. 2017; 58(8):4500. doi:

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Gene augmentation therapy in a large animal model of PDE6A -Retinitis Pigmentosa results in functional and structural rescue for at least 2.5 years.

Testing of a therapeutic vector for CNGB1 retinitis pigmentosa

The Contribution of Endoplasmic Reticulum Calcium Deficiency to Mistrafficking of Cone Outer Segment Proteins in CNG Channel Deficiency

The Role of Endoplasmic Reticulum (ER) Calcium Channels in ER Stress and Cone Death in CNG Channel Deficiency

Digenic triallelic inheritance in cone photoreceptor cyclic nucleotide gated channel associated retinopathies

Effects of Subretinal AAV8 Gene Therapy on Microperimetry in CNGA3 Achromatopsia Patients

Evaluation of reconstitution efficiencies of rAAV-dCas9-VPR split-intein protein trans-splicing vectors in retinal cells

Preclinical evaluation of rAAV.CNGB1 in the Cngb1 knockout mouse model of retinitis pigmentosa.

First Gene Supplementation Therapy for CNGA3-linked Achromatopsia

cGMP/PKG Signaling Regulation of Endoplasmic Reticulum Calcium Channels in Cyclic Nucleotide-gated Channel-deficient Cones

New insights into the mechanisms of retinal degeneration due to Phosphodiesterase 6 (PDE6) deficiency

Safety study in cynomolgus monkeys after subretinal injection of rAAV.hPDE6A

Humoral Immune Response After Intravitreal But Not After Subretinal AAV8 in Primates and Patients

Superior Retinal Gene Transfer and Biodistribution Profile of Subretinal Versus Intravitreal Delivery of AAV8 in Nonhuman Primates

Subretinal Injection for Gene Therapy Does Not Cause Clinically Significant Outer Nuclear Layer Thinning in Normal Primate Foveae

Peripherin-2 and Rom-1 Have Opposing Effects on Rod Outer Segment Targeting of Two Misfolded Peripherin-2 Mutants in Rods

Evaluation of electroretinography in cynomolgus monkeys after subretinal injection of rAAV.hPDE6A

Non-inferiority of subretinal versus intravitreal injection regarding photoreceptor layer thickness

Preclinical evaluation of rAAV8.CNGA3 in the Cnga3 knockout mouse model of ACHM2

Preclinical evaluation of rAAV8.PDE6A in the Pde6a D670G mutant mouse model of RP43

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