Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in rodent and swine photoreceptors
Shun-Yun Cheng, Jillian Caiazzi, Annabelle Biscans, Julia F. Alterman, Dimas Echeverria, Nicholas McHugh, Matthew R. Hassler, Samson M. Jolly, Delaney Giguere, Joris Cipi, Anastasia Khvorova, Claudio Punzo
This study evaluates tetravalent siRNA configurations for photoreceptor gene silencing, finding that tetravalent siRNA provides robust, long-lasting efficacy with a good safety profile in rodent and swine models.
Molecular Therapy — Nucleic Acids · Dec 5, 2023Peer-reviewedNeuroscience