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Anzahl der Publikationen: 4
Zeitschriftenartikel
Vignal-Clermont, Catherine; Yu-Wai-Man, Patrick; Newman, Nancy J.; Carelli, Valerio; Moster, Mark L.; Biousse, Valerie; Subramanian, Prem S.; Wang, An-Guor; Donahue, Sean P.; Leroy, Bart P.; Sadun, Alfredo A.; Klopstock, Thomas; Sergott, Robert C.; Fernandez, Gema Rebolleda; Chwalisz, Bart K.; Banik, Rudrani; Taiel, Magali; Roux, Michel und Sahel, Jose-Alain
(2023):
Safety of Lenadogene Nolparvovec Gene Therapy Over 5 Years in 189 Patients With Leber Hereditary Optic Neuropathy.
In: American Journal of Ophthalmology, Bd. 249: S. 108-125
[PDF, 1MB]
Carelli, Valerio; Newman, Nancy J.; Yu-Wai-Man, Patrick; Biousse, Valerie; Moster, Mark L.; Subramanian, Prem S.; Vignal-Clermont, Catherine; Wang, An-Guor; Donahue, Sean P.; Leroy, Bart P.; Sergott, Robert C.; Klopstock, Thomas; Sadun, Alfredo A.; Fernandez, Gema Rebolleda; Chwalisz, Bart K.; Banik, Rudrani; Girmens, Jean Francois; La Morgia, Chiara; DeBusk, Adam A.; Jurkute, Neringa; Priglinger, Claudia; Karanjia, Rustum; Josse, Constant; Salzmann, Julie; Montestruc, Francois; Roux, Michel; Taiel, Magali; Sahel, Jose-Alain und Group, The Lhon Study
(2022):
Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G > A MT-ND4 Mutation.
In: Ophthalmology and Therapy, Bd. 12, Nr. 1: S. 401-429
[PDF, 719kB]
Kuehlewein, Laura; Zobor, Ditta; Andreasson, Sten Olof; Ayuso, Carmen; Banfi, Sandro; Bocquet, Beatrice; Bernd, Antje S.; Biskup, Saskia; Boon, Camiel J. F.; Downes, Susan M.; Fischer, M. Dominik; Holz, Frank G.; Kellner, Ulrich; Leroy, Bart P.; Meunier, Isabelle; Nasser, Fadi; Rosenberg, Thomas; Rudolph, Gunther; Stingl, Katarina; Thiadens, Alberta A. H. J.; Wilhelm, Barbara; Wissinger, Bernd; Zrenner, Eberhart; Kohl, Susanne und Weisschuh, Nicole
(2020):
Clinical Phenotype and Course of PDE6A-Associated Retinitis Pigmentosa Disease, Characterized in Preparation for a Gene Supplementation Trial.
In: Jama Ophthalmology, Bd. 138, Nr. 12: S. 1241-1250
Ascari, Giulia; Peelman, Frank; Farinelli, Pietro; Rosseel, Toon; Lambrechts, Nina; Wunderlich, Kirsten A.; Wagner, Matias; Nikopoulos, Konstantinos; Martens, Pernille; Balikova, Irina; Derycke, Lara; Holtappels, Gabriele; Krysko, Olga; Laethem, Thalia van; De Jaegere, Sarah; Guillemyn, Brecht; De Rycke, Riet; De Bleecker, Jan; Creytens, David; Dorpe, Jo van; Gerris, Jan; Bachert, Claus; Neuhofer, Christiane; Walraedt, Sophie; Bischoff, Almut; Pedersen, Lotte B.; Klopstock, Thomas; Rivolta, Carlo; Leroy, Bart P.; De Baere, Elfride und Coppieters, Frauke
(2020):
Functional characterization of the first missense variant in CEP78, a founder allele associated with cone-rod dystrophy, hearing loss, and reduced male fertility.
In: Human Mutation, Bd. 41, Nr. 5: S. 998-1011
[PDF, 5MB]