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

• Main Authors: Antonio Maurizi (Author), Piergiorgio Patrizii (Author), Anna Teti (Author), Flavia Maria Sutera (Author), Paulina Baran-Rachwalska (Author), Chris Burns (Author), Uttom Nandi (Author), Michael Welsh (Author), Nissim Torabi-Pour (Author), Ashkan Dehsorkhi (Author), Suzanne Saffie-Siebert (Author)
• Format: Book
• Published: Elsevier, 2023-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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 li...