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International Journal of Molecular Sciences|June 2, 2021
Challenging Safety and Efficacy of Retinal Gene Therapies by RetinogenesisElena Marrocco, Rosa Maritato, Salvatore Botta, et al.
Brain : a Journal of Neurology|May 16, 2020
Gene replacement therapy provides benefit in an adult mouse model of Leigh syndromeRobin Reynaud-Dulaurier, Giorgia Benegiamo, Elena Marrocco, et al.
EMBO Molecular Medicine|January 27, 2011
Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosaClaudio Mussolino, Daniela Sanges, Elena Marrocco, et al.
Frontiers in Cell and Developmental Biology|March 21, 2020
Retinal Degeneration in MPS-IIIA Mouse ModelDaniela Intartaglia, Giuliana Giamundo, Elena Marrocco, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 3, 2024
Combined intraocular and intravenous gene delivery for therapy of gyrate atrophy of the choroid and retinaFabio Dell'Aquila, Roberto Di Cunto, Elena Marrocco, et al.
Plos One|August 31, 2013
Myosin7a deficiency results in reduced retinal activity which is improved by gene therapyPasqualina Colella, Andrea Sommella, Elena Marrocco, et al.
American Journal of Human Genetics|January 28, 2021
Allele-specific editing ameliorates dominant retinitis pigmentosa in a transgenic mouse modelClarissa Patrizi, Manel Llado, Daniela Benati, et al.
Molecular Therapy. Nucleic Acids|December 15, 2019
AAV-miR-204 Protects from Retinal Degeneration by Attenuation of Microglia Activation and Photoreceptor Cell DeathMarianthi Karali, Irene Guadagnino, Elena Marrocco, et al.
Molecular Therapy. Methods & Clinical Development|November 17, 2021
Inclusion of a degron reduces levelsof undesired inteins after AAV-mediated proteintrans-splicing in the retinaPatrizia Tornabene, Ivana Trapani, Miriam Centrulo, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|May 14, 2009
AAV-mediated tyrosinase gene transfer restores melanogenesis and retinal function in a model of oculo-cutaneous albinism type I (OCA1)Annagiusi Gargiulo, Ciro Bonetti, Sandro Montefusco, et al.
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