Sarepta Therapeutics - 엑손스키핑 대상 연구 확대

• Sarepta 치료학 - eteplirsen, 엑손 51 대상 엑손스키핑 약물을 개발하고 있습니다 - 추가 dystrophin의 exons 45, 50 잠재적인 DMD 치료로 53번을 타겟팅하는 계획을 발표했다.
• 엑손 53을 타겟팅하면 건너 뛰는 엑손을 통해 치료받을 수있는입니다. DMD에서 변이의 가장 널리 세트 중 하나를 해결합니다.
• Prosensa 추가 dystrophin의 exons 44, 45, 52, 잠재적 인 DMD 치료로 53과 55을 타겟팅 할 계획이다.

본문나가용.

Biopharmaceutical company Sarepta Therapeutics has announced it will expand the focus of its exon-skipping program for Duchenne muscular dystrophy (DMD) by developing compounds that target exons 45, 50 and 53 of the dystrophin gene, in addition to continuing to develop eteplirsen, which targets exon 51 of this gene.

Targeting exon 53, Sarepta says, will potentially address one of the most prevalent sets of mutations in DMD amenable to treatment via exon skipping: deletions of dystrophin exons 42-52, 45-52, 47-52, 48-52, 49-52, 50-52 or 52.

Currently, Sarepta’s experimental exon-skipping drug eteplirsen is undergoing clinical testing in boys with DMD, with very encouraging early results.

Dystrophin gene exons are sections of the gene that code for the dystrophin protein. (Dystrophin's absence from muscle tissue underlies DMD.) The experimental DMD treatment strategy known as exon skipping is based on laboratory studies showing that causing cells to ignore, or "skip," certain exons when making dystrophin protein can lead to production of functional, though shortened, dystrophin. Different dystrophin mutations require drugs that target different exons.

Sarepta, of Cambridge, Mass., announced its plans to develop new exon-skipping compounds in a Nov. 26, 2012, press release.

출처:http://quest.mda.org/news/dmd-sarepta-expands-exon-skipping-program