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Full-Text Articles in Polycyclic Compounds
Synthesis Of Small Molecules As Therapeutic Candidates, Savia Boyer
Synthesis Of Small Molecules As Therapeutic Candidates, Savia Boyer
Electronic Theses & Dissertations (2024 - present)
RNA plays a critical role in many biological functions, such as normal cellular functions or disease-related processes. Regulating biological processes through RNA-based therapeutics can be employed by the reversible photoregulation of oligonucleotides. Azobenzene, a light-responsive compound that changes conformation when exposed to ultraviolet (UV) or visible light, is attached to cytidine phosphoramidite to be incorporated in a series of RNA oligonucleotides. The reversibility of the nucleoside and oligonucleotide is verified through UV melting point experiments. The modified oligonucleotide can change conformation upon light irradiation to stop reverse transcription (RT) in the presence of many different reverse transcriptase enzymes. This technique …
Advancing Small Molecule Therapies For Myotonic Dystrophy Type 1, Sawyer M. Hicks
Advancing Small Molecule Therapies For Myotonic Dystrophy Type 1, Sawyer M. Hicks
Electronic Theses & Dissertations (2024 - present)
Myotonic dystrophy type 1 (DM1) is a multisystemic disorder caused by the expression of expanded CUG (CUGexp) repeat RNA from the myotonic dystrophy protein kinase (DMPK) gene. This CUGexp repeat RNA’s gain-of-function mechanism leads to the sequestration of muscleblind-like (MBNL) proteins, resulting in widespread alternative splicing dysregulation across tissues. Despite significant research, there are currently no approved therapeutics targeting the underlying causes of DM1. This dissertation explores the development and optimization of a novel class of small molecules, Modified Polycyclic Compounds (MPCs), designed to address splicing dysregulation in DM1. MPCs were designed from previously published …