PRMT inhibitor promotes SMN2 exon 7 inclusion and synergizes with nusinersen to rescue SMA mice

Authors: Anna J Kordala, Jessica Stoodley, Nina Ahlskog, Muhammad Hanifi, Antonio Garcia Guerra, Amarjit Bhomra, Wooi Fang Lim, Lyndsay M Murray, Kevin Talbot, Suzan M Hammond, Matthew JA Wood, Carlo Rinaldi

Source: EMBO Molecular Medicine, Vol 15, Iss 11, Pp 1-16 (2023)

Subject Terms: nusinersen, PRMT inhibitor, small molecule, spinal muscular atrophy, Medicine (General), R5-920, Genetics, QH426-470

File Description: electronic resource

Relation: https://doaj.org/toc/1757-4676; https://doaj.org/toc/1757-4684

Access URL: https://doaj.org/article/0a6be327d14849d1ae2a1fc49bf73886

Antibody-oligonucleotide conjugate achieves CNS delivery in animal models for spinal muscular atrophy

Authors: Suzan M. Hammond, Frank Abendroth, Larissa Goli, Jessica Stoodley, Matthew Burrell, George Thom, Ian Gurrell, Nina Ahlskog, Michael J. Gait, Matthew J.A. Wood, Carl I. Webster

Source: JCI Insight, Vol 7, Iss 24 (2022)

Subject Terms: Neuroscience, Medicine

File Description: electronic resource

Relation: https://doaj.org/toc/2379-3708

Access URL: https://doaj.org/article/e10b5264221549b9be42dfc52acf597f