Identification of potent siRNA targeting complement C5 and its robust activity in pre-clinical models of myasthenia gravis and collagen-induced arthritis

• Main Authors: Yoshikazu Kuboi (Author), Yuta Suzuki (Author), Sotaro Motoi (Author), Chiyuki Matsui (Author), Naoki Toritsuka (Author), Tomoya Nakatani (Author), Kazuhiro Tahara (Author), Yoshinori Takahashi (Author), Yoko Ida (Author), Ayaka Tomimatsu (Author), Motohiro Soejima (Author), Toshio Imai (Author)
• Format: Book
• Published: Elsevier, 2023-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Complement component 5 (C5), an important molecule in the complement cascade, blockade by antibodies shows clinical efficacy in treating complement-mediated disorders. However, insufficient blockading induced by single-nucleotide polymorphisms in the C5 protein or frequent development of "break...