Antisense oligonucleotides and spinal muscular atrophy: skipping along.
نویسندگان
چکیده
Antisense oligonucleotides (ASOs) can be used to alter the splicing of a gene and either restore production of a required protein or eliminate a toxic product. In this issue of Genes & Development, Hua and colleagues (pp. 1634-1644) show that ASOs directed against an intron splice silencer (ISS) in the survival motor neuron 2 (SMN2) gene alter the amount of full-length SMN transcript in the nervous system, restoring SMN to levels that could correct spinal muscular atrophy (SMA).
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ورودعنوان ژورنال:
- Genes & development
دوره 24 15 شماره
صفحات -
تاریخ انتشار 2010