51�Թ�

An additional trawl through genetic databases and the morpholino literature revealed that 80% of results from published morpholino experiments were not reproducible in genetic mutants2.

Using this animal model, we have developed an antisense morpholino–based therapy that prevents the pathogenic Lmna splicing, markedly reducing the accumulation of progerin and its associated nuclear defects.

Writing in the journal Nature Medicine, Sina Bavari and colleagues said the drugs tested are antisense phosphorodiamidate morpholino oligomers, or PMOs, called AVI-6002 and AVI-6003.