Long-term reversal of Duchenne muscular dystrophy via circular arRNA-guided exon skipping in monkeys and humans

2026/06/10 by Wenting Guo, Huixian Tang, Zongyi Yi +36 · 1 voice
Biochemistry, Genetics and Molecular Biology · #CRISPR and Genetic Engineering #Muscle Physiology and Disorders #RNA regulation and disease

paper · doi:10.1016/j.cell.2026.05.030

openalex publication_date 2026/06/10 · crossref created 2026/06/10 · openalex created_date 2026/06/11 · crossref issued 2026/07/01 · crossref published 2026/07/01 · crossref published-print 2026/07/01 · crossref deposited 2026/07/09 · crossref indexed 2026/07/10 · openalex updated_date 2026/07/12

Citations

• The dynamic epitranscriptome: A to I editing modulates genetic information
• Satellite Cells and the Muscle Stem Cell Niche

Discussions

• DMD is one of the most challenging diseases in gene therapy. Wensheng Wei' s lab unveils a novel exon skipping strategy based on LEAPER 2.0. Using circular ADAR‑recruiting RNA, it demonstrates safe, l [bsky, 0 points, 0 comments]