Anzahl der Publikationen: 12
Zeitschriftenartikel
Reichel, Felix Friedrich ORCID: https://orcid.org/0000-0001-9325-5616; Michalakis, Stylianos ORCID: https://orcid.org/0000-0001-5092-9238; Wilhelm, Barbara; Zobor, Ditta; Muehlfriedel, Regine; Kohl, Susanne; Weisschuh, Nicole; Sothilingam, Vithiyanjali; Kuehlewein, Laura ORCID: https://orcid.org/0000-0003-1906-8681; Kahle, Nadine; Seitz, Immanuel ORCID: https://orcid.org/0000-0002-3086-6425; Paquet-Durand, Francois ORCID: https://orcid.org/0000-0001-7355-5742; Tsang, Stephen H.; Martus, Peter; Peters, Tobias; Seeliger, Mathias; Bartz-Schmidt, Karl Ulrich; Ueffing, Marius; Zrenner, Eberhard; Biel, Martin; Wissinger, Bernd und Fischer, Dominik
(2021):
Three-year results of phase I retinal gene therapy trial for CNGA3-mutated achromatopsia: results of a non randomised controlled trial.
In: British Journal of Ophthalmology, Bd. 106, Nr. 11: S. 1567-1572
Fischer, M. Dominik; Michalakis, Stylianos; Wilhelm, Barbara; Zobor, Ditta; Muehlfriedel, Regine; Kohl, Susanne; Weisschuh, Nicole; Ochakovski, G. Alex; Klein, Reinhild; Schoen, Christian; Sothilingam, Vithiyanjali; Garcia-Garrido, Marina; Kuehlewein, Laura; Kahle, Nadine; Werner, Annette; Dauletbekov, Daniyar; Paquet-Durand, Francois; Tsang, Stephen; Martus, Peter; Peters, Tobias; Seeliger, Mathias; Bartz-Schmidt, Karl Ulrich; Ueffing, Marius; Zrenner, Eberhart; Biel, Martin und Wissinger, Bernd
(2020):
Safety and Vision Outcomes of Subretinal Gene Therapy Targeting Cone Photoreceptors in Achromatopsia A Nonrandomized Controlled Trial.
In: Jama Ophthalmology, Bd. 138, Nr. 6: S. 643-651
Tobias, Peters; Philipp, Seitz Immanuel; Stylianos, Michalakis; Martin, Biel; Barbara, Wilhelm; Felix, Reichel; Alexander, Ochakovski Guy; Eberhart, Zrenner; Marius, Ueffing; Birgit, Korbmacher; Sven, Korte; Ulrich, Bartz-Schmidt Karl; Dominik, Fischer Manuel; Bartz-Schmidt, Karl Ulrich; Bolz, Sylvia; Fischer, Dominik; Kohl, Susanne; Kuehlewein, Laura; Muehlfriedel, Regine; Neubauer, Jonas; Ochakovski, Alex; Paquet-Durand, Francois; Seeliger, Mathias; Sothilingam, Vithiyanjali; Ueffing, Marius; Weisschuh, Nicole; Wissinger, Bernd; Zhour, Ahmad; Zobor, Ditta; Zrenner, Eberhart; Biel, Martin; Michalakis, Stylianos; Schön, Christian; Kahle, Nadine; Peters, Tobias; Wilhelm, Barbara; Tsang, Steven und Gloeckner, Christian Johannes
(2019):
Safety and Toxicology of Ocular Gene Therapy with Recombinant AAV Vector rAAV.hCNGA3 in Nonhuman Primates.
In: Human Gene Therapy Clinical Development, Bd. 30, Nr. 2: S. 50-56
Burkard, Markus; Kohl, Susanne; Kratzig, Timm; Tanimoto, Naoyuki; Brennenstuhl, Christina; Bausch, Anne E.; Junger, Katrin; Reuter, Peggy; Sothilingam, Vithiyanjali; Beck, Susanne C.; Huber, Gesine; Ding, Xi-Qin; Mayer, Anja K.; Baumann, Britta; Weisschuh, Nicole; Zobor, Ditta; Hahn, Gesa-Astrid; Kellner, Ulrich; Venturelli, Sascha; Becirovic, Elvir; Charbel Issa, Peter; Koenekoop, Robert K.; Rudolph, Gunther; Heckenlively, John; Sieving, Paul; Weleber, Richard G.; Hamel, Christian; Zong, Xiangang; Biel, Martin; Lukowski, Robert; Seeliger, Matthias W.; Michalakis, Stylianos; Wissinger, Bernd und Ruth, Peter
(2018):
Accessory heterozygous mutations in cone photoreceptor CNGA3 exacerbate CNG channel-associated retinopathy.
In: The Journal of Clinical Investigation, Bd. 128, Nr. 12: S. 5663-5675
Kahle, Nadine A.; Peters, Tobias; Zobor, Ditta; Kuehlewein, Laura; Kohl, Susanne; Zhour, Ahmad; Werner, Annette; Seitz, Immanuel P.; Sothilingam, Vithiyanjali; Michalakis, Stylianos; Biel, Martin; Ueffing, Marius; Zrenner, Eberhart; Bartz-Schmidt, Karl U.; Fischer, M. Dominik und Wilhelm, Barbara J. C.
(2018):
Development of Methodology and Study Protocol: Safety and Efficacy of a Single Subretinal Injection of rAAV.hCNGA3 in Patients with CNGA3-Linked Achromatopsia Investigated in an Exploratory Dose-Escalation Trial.
In: Human Gene Therapy Clinical Development, Bd. 29, Nr. 3: S. 121-131
Schön, Christian; Sothilingam, Vithiyanjali; Mühlfriedel, Regine; Garrido, Marina Garcia; Beck, Susanne C.; Tanimoto, Naoyuki; Wissinger, Bernd; Paquet-Durand, Francois; Biel, Martin; Michalakis, Stylianos und Seeliger, Mathias W.
(2017):
Gene Therapy Successfully Delays Degeneration in a Mouse Model of PDE6A-Linked Retinitis Pigmentosa (RP43).
In: Human Gene therapy, Bd. 28, Nr. 12: S. 1180-1188
Michalakis, Stylianos; Fischer, M. Dominik; Schön, Christian; Wilhelm, Barbara; Zobor, Ditta; Muhlfriedel, Regine; Sothilingam, Vithiyanjali; Kohl, Susanne; Peters, Tobias; Zrenner, Eberhart; Bartz-Schmidt, Karl Ulrich; Üffing, Marius; Wissinger, Bernd; Seeliger, Mathias und Biel, Martin
(2017):
Gene Supplementation Therapy for CNGA3-Linked Achromatopsia.
In: Molecular therapy, Bd. 25, Nr. 5: S. 354
Mühlfriedel, Regine; Tanimoto, Naoyuki; Schön, Christian; Sothilingam, Vithiyanjali; Garrido, Marina Garcia; Beck, Susanne C.; Huber, Gesine; Biel, Martin; Seeliger, Mathias W. und Michalakis, Stylianos
(2017):
AAV-Mediated Gene Supplementation Therapy in Achromatopsia Type 2: Preclinical Data on Therapeutic Time Window and Long-Term Effects.
In: Frontiers in Neuroscience, Bd. 11, 292
[PDF, 5MB]
Michalakis, Stylianos; Shaltiel, Lior; Sothilingam, Vithiyanjali; Koch, Susanne; Schludi, Verena; Krause, Stefanie; Zeitz, Christina; Audo, Isabelle; Lancelot, Marie-Elise; Hamel, Christian; Meunier, Isabelle; Preising, Markus N.; Friedburg, Christoph; Lorenz, Birgit; Zabouri, Nawal; Haverkamp, Silke; Garcia Garrido, Marina; Tanimoto, Naoyuki; Seeliger, Mathias W.; Biel, Martin und Wahl-Schott, Christian A.
(2017):
Mosaic synaptopathy and functional defects in Cav1.4 heterozygous mice and human carriers of CSNB2 (vol 23, pg 1538, 2014).
In: Human Molecular Genetics, Bd. 26, Nr. 2: S. 466
Schön, Christian; Asteriti, Sabrina; Koch, Susanne; Sothilingam, Vithiyanjali; Garrido, Marina Garcia; Tanimoto, Naoyuki; Herms, Jochen; Seeliger, Mathias W.; Cangiano, Lorenzo; Biel, Martin und Michalakis, Stylianos
(2016):
Loss of HCN1 enhances disease progression in mouse models of CNG channel-linked retinitis pigmentosa and achromatopsia.
In: Human Molecular Genetics, Bd. 25, Nr. 6: S. 1165-1175
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