Therapeutic phosphorodiamidate morpholino oligonucleotides: Physical properties, solution structures, and folding thermodynamics

• Main Authors: Farkhad Maksudov (Author), Evgenii Kliuchnikov (Author), Daniel Pierson (Author), M.L. Ujwal (Author), Kenneth A. Marx (Author), Arani Chanda (Author), Valeri Barsegov (Author)
• Format: Book
• Published: Elsevier, 2023-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Elucidating the structure-function relationships for therapeutic RNA mimicking phosphorodiamidate morpholino oligonucleotides (PMOs) is challenging due to the lack of information about their structures. While PMOs have been approved by the US Food and Drug Administration for treatment of Duchenne mu...