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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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10480457/
https://www.ncbi.nlm.nih.gov/pubmed/37680985
http://dx.doi.org/10.1016/j.omtn.2023.08.020

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10480457/
https://www.ncbi.nlm.nih.gov/pubmed/37680985
http://dx.doi.org/10.1016/j.omtn.2023.08.020

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