Summary Antisense therapeutic oligonucleotides are entering a transformative era in clinical development, with a total of 21 oligonucleotides approved and a robust pipeline poised for the coming years. These drugs have emerged as a cornerstone of genetic medicine, providing a programmable platform to modulate gene expr...
Bruno Miguel Da Cruz Godinho, Chantal M. Ferguson, Annemieke Aartsma-Rus· Cell Reports Medicine· 0 citations
“Sharing what works, what does not, what is safe, and what is not would advance the field considerably…”Jokūbas Leikauskas, Editor, Nucleic Acid Insights , speaks with Annemieke Aartsma-Rus, Professor of Translational Genetics, Leiden University Medical Center, about why delivery remains the central challenge in exon s...
Evidence of isoform restoration, immune activation following treatment, and a framework to evaluate exon-skipping therapies in the CNS using spatial transcriptomics are presented.
Qirong Mao, A. Ahmadi, S. D. de Vries et al.· iScience· 0 citations
We use cookies to run the site and, with your consent, for analytics and to show ads.
See our Cookie Policy.
