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drisapersen
Drisapersen (also known as Kyndrisa, PRO051 and GSK2402968〔(【引用サイトリンク】url=http://www.prosensa.eu/technology-and-products/pipeline/pro051gsk2402968 )〕) is an experimental drug that is under development by BioMarin, after acquisition of Prosensa, for the treatment of Duchenne muscular dystrophy. The drug is a 2'-O-methyl phosphorothioate oligonucleotide that alters the splicing of the dystrophin RNA transcript, eliminating exon 51 from the mature dystrophin mRNA.
==Mechanism of action==
Duchenne muscular dystrophy (DMD) is caused when a mutation in the DMD gene changes the DMD RNA so that it no longer codes for functional dystrophin protein, usually due to a mutation that alters the reading frame of the RNA downstream of the mutation. If an exon with an appropriate number of bases lies near the mutation, by removing that exon the downstream reading frame can be corrected and production of partially functional dystrophin can be restored. This is the general strategy used for designing exon-skipping oligonucleotides for DMD; as there are 79 exons in the longest splice form of the dystrophin transcript, many different oligos are needed to address the range of mutations present in the population of people with DMD.
抄文引用元・出典: フリー百科事典『 ウィキペディア（Wikipedia）』
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