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학술연구/단체지원/교육 등 연구자 활동을 지속하도록 DBpia가 지원하고 있어요.
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연구자들이 자신의 연구와 전문성을 널리 알리고, 새로운 협력의 기회를 만들 수 있는 네트워킹 공간이에요.
논문 기본 정보
- 자료유형
- 학술저널
- 저자정보
- 발행연도
- 2020.1
- 수록면
- 273 - 283 (11page)
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초록· 키워드
오류제보하기
The reduction of survival motor neuron (SMN) protein causes spinal muscular atrophy (SMA), an autosomal recessive neuromuscular disease. Nusinersen is an antisense oligonucleotide, approved by the FDA, which specifically binds to the repressor within SMN2 exon 7 to enhance exon 7 inclusion and augment production of functional SMN protein. Nusinersen is the first new oligonucleotide-based drug targeting the central nervous system for the treatment of SMA. This review of nusinersen will discuss its action mechanism, cellular uptake, trafficking mechanisms, and administration approaches to cross the blood-brain barrier. Furthermore, nusinersen clinical trials will be assessed in terms of pharmacokinetics, tolerability and safety, the clinical outcomes of multiple intrathecal doses, and a discussion on the primary and secondary endpoints.
목차
등록된 정보가 없습니다.
참고문헌 (82)
참고문헌 신청
Hua Y / 2010 / Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model / Genes Dev 24:1634~1644
Singh NK / 2006 / Splicing of a critical exon of human Survival Motor Neuron is regulated by a unique silencer element located in the last intron / Mol Cell Biol 26:1333~1346
Wan L / 2005 / The survival of motor neurons protein determines the capacity for snRNP assembly : biochemical deficiency in spinal muscular atrophy / Mol Cell Biol 25:5543~5551
Hua Y / 2007 / Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon / PLoS Biol 5:e73
Kashima T / 2003 / A negative element in SMN2 exon 7 inhibits splicing in spinal muscular atrophy / Nat Genet 34:460~463
Singh NK / 2006 / Splicing of a critical exon of human Survival Motor Neuron is regulated by a unique silencer element located in the last intron / Mol Cell Biol 26:1333~1346
Wan L / 2005 / The survival of motor neurons protein determines the capacity for snRNP assembly : biochemical deficiency in spinal muscular atrophy / Mol Cell Biol 25:5543~5551
Hua Y / 2007 / Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon / PLoS Biol 5:e73
Kashima T / 2003 / A negative element in SMN2 exon 7 inhibits splicing in spinal muscular atrophy / Nat Genet 34:460~463
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