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Investigative Ophthalmology & Visual Science|April 9, 2011
Evaluation of Italian patients with leber congenital amaurosis due to AIPL1 mutations highlights the potential applicability of gene therapyFrancesco Testa, Enrico Maria Surace, Settimio Rossi, et al.Scientific Reports|December 7, 2017
MiR-211 is essential for adult cone photoreceptor maintenance and visual functionSara Barbato, Elena Marrocco, Daniela Intartaglia, et al.EMBO Molecular Medicine|October 4, 2022
miR-181a/b downregulation: a mutation-independent therapeutic approach for inherited retinal diseasesSabrina Carrella, Martina Di Guida, Simona Brillante, et al.The EMBO Journal|March 11, 2020
Light-responsive microRNA miR-211 targets Ezrin to modulate lysosomal biogenesis and retinal cell clearanceFederica Naso, Daniela Intartaglia, Danila Falanga, et al.Nature Communications|June 10, 2021
Altered heparan sulfate metabolism during development triggers dopamine-dependent autistic-behaviours in models of lysosomal storage disordersMaria De Risi, Michele Tufano, Filomena Grazia Alvino, et al.EMBO Molecular Medicine|April 14, 2019
miR-181a/b downregulation exerts a protective action on mitochondrial disease modelsAlessia Indrieri, Sabrina Carrella, Alessia Romano, et al.Nature Communications|April 13, 2022
Therapeutic homology-independent targeted integration in retina and liverPatrizia Tornabene, Rita Ferla, Manel Llado-Santaeularia, et al.Science Translational Medicine|May 17, 2019
Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retinaPatrizia Tornabene, Ivana Trapani, Renato Minopoli, et al.Pageof 3