Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson–Gilford progeria syndrome

Recurrent de novo point mutations in lamin A cause Hutchinson–Gilford progeria syndrome

Maria Eriksson, W. Ted Brown, Leslie B. Gordon et al. · Nature · 2003 · 2.2K citations · Full text

Medicine, Genetics, Molecular Genetics +5

Lamin A Truncation in Hutchinson-Gilford Progeria

Annachiara De Sandre‐Giovannoli, Rafaëlle Bernard, Pierre Cau et al. · Science · 2003 · 1.5K citations

Life Sciences, Anatomy, Pteridology +4

Reversal of the cellular phenotype in the premature aging disease Hutchinson-Gilford progeria syndrome

Paola Scaffidi, Tom Misteli · Nature Medicine · 2005 · 621 citations · Full text

Education, Genetics, Molecular Genetics +10

Identification of Long-Lived Proteins Reveals Exceptional Stability of Essential Cellular Structures

Brandon H. Toyama, Jeffrey N. Savas, Sung Kyu Park et al. · Cell · 2013 · 604 citations · Full text

Life Sciences, Cell Biology, Molecular Biology +14

Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

Marco A. Passini, Jie Bu, Amy M. Richards et al. · Science Translational Medicine · 2011 · 553 citations · Full text

Medicine, Neurobiology, Neuroscience +12