Novel hybrid silicon-lipid nanoparticles deliver a siRNA to cure autosomal dominant osteopetrosis in mice. Implications for gene therapy in humans

Novel hybrid silicon-lipid nanoparticles deliver a siRNA to cure autosomal dominant osteopetrosis in mice. Implications for gene therapy in humans

Rare skeletal diseases are still in need of proper clinically available transfection agents as the major challenge for first-in-human translation relates to intrinsic difficulty in targeting bone without exacerbating any inherent toxicity due to used vector. SiSaf’s silicon stabilized hybrid lipid n...

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Bibliographic Details
Main Authors: Antonio Maurizi, Piergiorgio Patrizii, Anna Teti, Flavia Maria Sutera, Paulina Baran-Rachwalska, Chris Burns, Uttom Nandi, Michael Welsh, Nissim Torabi-Pour, Ashkan Dehsorkhi, Suzanne Saffie-Siebert
Format: Article
Language:English
Published: Elsevier 2023-09-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: delivery strategies
therapy
genetic bone diseases
autosomal dominant osteopetrosis
bone resorption
osteoclasts
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253123002317

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http://www.sciencedirect.com/science/article/pii/S2162253123002317

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