Effective In Vivo Topical Delivery of siRNA and Gene Silencing in Intact Corneal Epithelium Using a Modified Cell-Penetrating Peptide

• Main Authors: Davide Schiroli (Author), María J. Gómara (Author), Eleonora Maurizi (Author), Sarah D. Atkinson (Author), Laura Mairs (Author), Kathleen A. Christie (Author), Diego F. Cobice (Author), Cian M. McCrudden (Author), M. Andrew Nesbit (Author), Isabel Haro (Author), Tara Moore (Author)
• Format: Book
• Published: Elsevier, 2019-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Autosomal dominantly inherited genetic disorders such as corneal dystrophies are amenable to allele-specific gene silencing with small interfering RNA (siRNA). siRNA delivered to the cornea by injection, although effective, is not suitable for a frequent long-term treatment regimen, whereas topical...