Activating the synthesis of progerin, the mutant prelamin A in Hutchinson–Gilford progeria syndrome, with antisense oligonucleotides

@article{Fong2009ActivatingTS,
  title={Activating the synthesis of progerin, the mutant prelamin A in Hutchinson–Gilford progeria syndrome, with antisense oligonucleotides},
  author={Loren G. Fong and Timothy A. Vickers and Emily A. Farber and Christine E Choi and Ui Jeong Yun and Yan Hu and Shao H. Yang and Catherine Coffinier and Roger Lee and Liya Yin and Brandon S.J. Davies and Douglas A. Andres and Hans Peter Spielmann and C. F. Bennett and Stephen G. Young},
  journal={Human Molecular Genetics},
  year={2009},
  volume={18},
  pages={2462 - 2471}
}
Hutchinson-Gilford progeria syndrome (HGPS) is caused by point mutations that increase utilization of an alternate splice donor site in exon 11 of LMNA (the gene encoding lamin C and prelamin A). The alternate splicing reduces transcripts for wild-type prelamin A and increases transcripts for a truncated prelamin A (progerin). Here, we show that antisense oligonucleotides (ASOs) against exon 11 sequences downstream from the exon 11 splice donor site promote alternate splicing in both wild-type… CONTINUE READING