Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in mouse and pig photoreceptors

• Main Authors: Shun-Yun Cheng (Author), Jillian Caiazzi (Author), Annabelle Biscans (Author), Julia F. Alterman (Author), Dimas Echeverria (Author), Nicholas McHugh (Author), Matthew Hassler (Author), Samson Jolly (Author), Delaney Giguere (Author), Joris Cipi (Author), Anastasia Khvorova (Author), Claudio Punzo (Author)
• Format: Book
• Published: Elsevier, 2024-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Inherited retinal dystrophies caused by dominant mutations in photoreceptor (PR) cell expressed genes are a major cause of irreversible vision loss. Oligonucleotide therapy has been of interest in diseases that conventional medicine cannot target. In the early days, small interfering RNAs (siRNAs) w...