1044243-51-7Relevant articles and documents
EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPY
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Paragraph 0346; 0348, (2020/07/07)
Antisense oligomers complementary to a selected target site in the human dystrophin gene to induce exon 50 skipping are described. In various aspects, antisense oligomers are described according to Formula (I): or a pharmaceutically acceptable salt thereo
Antisense-induced exon exclusion in type VII collagen
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Page/Page column 73, (2020/12/13)
The present disclosure relates to antisense oligomers and related compositions and methods for increasing the expression of functional human type VII collagen and methods for treating dystrophic epidermolysis bullosa and related disorders and relates to inducing exclusion of exon 80 in human type VII collagen mRNA.
PROCESSES FOR PREPARING PHOSPHORODIAMIDATE MORPHOLINO OLIGOMERS VIA FAST-FLOW SYNTHESIS
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Page/Page column 86-87, (2019/04/16)
Provided herein are processes for preparing an oligomer (e.g., a morpholino oligomer). The synthetic processes described herein may be advantageous to scaling up oligomer synthesis while maintaining overall yield and purity of a synthesized oligomer.
EXON SKIPPING OLIGOMERS AND OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
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Page/Page column 133-134, (2020/01/08)
Antisense oligomers and antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.
EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
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Page/Page column 86-88, (2018/07/22)
Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.
EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
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Paragraph 0309; 0311, (2018/07/15)
Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 51 skipping are described.
EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
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Page/Page column 86; 87, (2018/07/29)
Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 45 skipping are described.
PROCESSES FOR PREPARING OLIGOMERS
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Page/Page column 161, (2017/12/29)
Provided herein are processes for preparing an oligomer (e.g., a morpholino oligomer). The synthetic processes described herein may be advantageous to scaling up oligomersynthesis while maintaining overall yield and purity of a synthesized oligomer.
COMPOSITIONS AND METHODS FOR TREATING DUCHENNE MUSCULAR DYSTROPHY AND RELATED DISORDERS
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Paragraph 00424, (2017/05/14)
The present disclosure relates to compositions and methods for the treatment of Duchenne muscular dystrophy and related disorders. Modified antisense oligomers are disclosed for the treatment of Duchenne muscular dystrophy and related disorders.
EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
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Paragraph 0250, (2016/05/02)
Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.