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Hutchinson-Gilford Progeria Syndrome (HGPS) is a disease caused by a single nucleotide mutation in the Lamin A gene (LMNA), which is characterized by accelerated aging, defects in the cardiovascular system, and premature death.
At present, the only treatment option for HGPS is the recently approved lonafarnib, but the drug has treatment limitations such as side effects.
The most common pathogenic mutation in the LMNA gene is C1824T, which leads to abnormal pre-mRNA splicing, leading to the production of progerin protein, which destroys normal cell nuclear structure and function, and ultimately causes disease.
In one study, researchers used antisense phosphorodiamidate oligomers (PMO) to prevent RNA splicing: these synthetic nucleotide analogs can prevent the translation of the target sequence and prevent the spliceosome from interacting with the mRNA.
In another study, the researchers designed and screened 198 ASOs with different target sequences, lengths and chemical properties for fibroblasts derived from patients.
Two studies have shown that ASO can be delivered to key organs affected by HGPS, thereby reducing the expression of progerin protein and extending the life of mice.
Reference materials:
[1] Erdos, MR, Cabral, WA, Tavarez, UL et al.
[2] Puttaraju, M.