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Erdos, Michael R; Cabral, Wayne A; Tavarez, Urraca L; Cao, Kan; Gvozdenovic-Jeremic, Jelena; Narisu, Narisu; Zerfas, Patricia M; Crumley, Stacy; Boku, Yoseph; Hanson, Gunnar; Mourich, Dan V; Kole, Ryszard; Eckhaus, Michael A; Gordon, Leslie B; Collins, Francis S
Nature medicine, 03/2021, Letnik: 27, Številka: 3Journal Article
Hutchinson-Gilford progeria syndrome (HGPS) is a rare accelerated aging disorder characterized by premature death from myocardial infarction or stroke. It is caused by de novo single-nucleotide mutations in the LMNA gene that activate a cryptic splice donor site, resulting in the production of a toxic form of lamin A, which is termed progerin. Here we present a potential genetic therapeutic strategy that utilizes antisense peptide-conjugated phosphorodiamidate morpholino oligomers (PPMOs) to block pathogenic splicing of mutant transcripts. Of several candidates, PPMO SRP-2001 provided the most significant decrease in progerin transcripts in patient fibroblasts. Intravenous delivery of SRP-2001 to a transgenic mouse model of HGPS produced significant reduction of progerin transcripts in the aorta, a particularly critical target tissue in HGPS. Long-term continuous treatment with SRP-2001 yielded a 61.6% increase in lifespan and rescue of vascular smooth muscle cell loss in large arteries. These results provide a rationale for proceeding to human trials.
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in: SICRIS
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