TY  - RPRT
TI  - Peptide-phosphorodiamidate morpholino oligomer therapy for dysferlinopathy induces pseudoexon skipping and restoration of functional protein.
AU  - James E Gooding
AU  - Gyeongsu Park
AU  - Atish Wagh
AU  - Jonathan K Watts
AU  - Janice A Dominov
AU  - Robert H Brown
PY  - 2026
DO  - 10.1172/jci.insight.204742
UR  - https://pubmed.ncbi.nlm.nih.gov/42446943/
ID  - 42446943
ER  -