Novel hybrid silicon-lipid nanoparticles deliver a siRNA to cure autosomal dominant osteopetrosis in mice. Implications for gene therapy in humans
Maurizi, Antonio, Patrizii, Piergiorgio, Teti, Anna, Sutera, Flavia Maria, Baran-Rachwalska, Paulina, Burns, Chris, Nandi, Uttom, Welsh, Michael, Torabi-Pour, Nissim, Dehsorkhi, Ashkan, Saffie-Siebert, Suzanne
Published in Molecular therapy. Nucleic acids (12.09.2023)
Published in Molecular therapy. Nucleic acids (12.09.2023)
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Journal Article
Development of a potential clinical product for treating CLCN7-dependent Autosomal Dominant Osteopetrosis type 2 (ADO2)
Maurizi, Antonio, Patrizii, Piergiorgio, Teti, Anna, Sutera, Flavia Maria, Hadianamrei, Roja, Dehsorkhi, Ashkan, Saffie-Siebert, Suzanne
Published in Bone Reports (01.05.2022)
Published in Bone Reports (01.05.2022)
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Journal Article