Strategies for In Vivo Screening and Mitigation of Hepatotoxicity Associated with Antisense Drugs

• Main Authors: Piotr J. Kamola (Author), Klio Maratou (Author), Paul A. Wilson (Author), Kay Rush (Author), Tanya Mullaney (Author), Tom McKevitt (Author), Paula Evans (Author), Jim Ridings (Author), Probash Chowdhury (Author), Aude Roulois (Author), Ann Fairchild (Author), Sean McCawley (Author), Karen Cartwright (Author), Nigel J. Gooderham (Author), Timothy W. Gant (Author), Kitty Moores (Author), Stephen A. Hughes (Author), Mark R. Edbrooke (Author), Kenneth Clark (Author), Joel D. Parry (Author)
• Format: Book
• Published: Elsevier, 2017-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Antisense oligonucleotide (ASO) gapmers downregulate gene expression by inducing enzyme-dependent degradation of targeted RNA and represent a promising therapeutic platform for addressing previously undruggable genes. Unfortunately, their therapeutic application, particularly that of the more potent...