Ontology highlight
ABSTRACT:
SUBMITTER: Kandasamy P
PROVIDER: S-EPMC9178015 | biostudies-literature | 2022 Jun
REPOSITORIES: biostudies-literature
Kandasamy Pachamuthu P McClorey Graham G Shimizu Mamoru M Kothari Nayantara N Alam Rowshon R Iwamoto Naoki N Kumarasamy Jayakanthan J Bommineni Gopal R GR Bezigian Adam A Chivatakarn Onanong O Butler David C D DCD Byrne Michael M Chwalenia Katarzyna K Davies Kay E KE Desai Jigar J Shelke Juili Dilip JD Durbin Ann F AF Ellerington Ruth R Edwards Ben B Godfrey Jack J Hoss Andrew A Liu Fangjun F Longo Kenneth K Lu Genliang G Marappan Subramanian S Oieni Jacopo J Paik Ik-Hyeon IH Estabrook Erin Purcell EP Shivalila Chikdu C Tischbein Maeve M Kawamoto Tomomi T Rinaldi Carlo C Rinaldi Carlo C Rajão-Saraiva Joana J Tripathi Snehlata S Yang Hailin H Yin Yuan Y Zhao Xiansi X Zhou Cong C Zhang Jason J Apponi Luciano L Wood Matthew J A MJA Vargeese Chandra C
Nucleic acids research 20220601 10
Although recent regulatory approval of splice-switching oligonucleotides (SSOs) for the treatment of neuromuscular disease such as Duchenne muscular dystrophy has been an advance for the splice-switching field, current SSO chemistries have shown limited clinical benefit due to poor pharmacology. To overcome limitations of existing technologies, we engineered chimeric stereopure oligonucleotides with phosphorothioate (PS) and phosphoryl guanidine-containing (PN) backbones. We demonstrate that the ...[more]