Oligomers

Details Oligomers Oligomers

2009-09-10

2011-12-27

Chong, Kimberly (Department: 1635)

Organic compounds -- part of the class 532-570 series

Organic compounds

Carbohydrates or derivatives

C536S024310, C536S024100, C514S04400A

Reexamination Certificate

active

08084601

ABSTRACT:
Molecules are provided for inducing or facilitating exon skipping in forming spliced mRNA products from pre-mRNA molecules in cells. The molecules may be provided directly as oligonucleotides or expression products of vectors that are administered to a subject. High rates of skipping can be achieved. High rates of skipping reduce the severity of a disease like Duchene Muscular Dystrophy so that the disease is more like Becker Muscular Dystrophy. This is a severe reduction in symptom severity and mortality.

REFERENCES:
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patent: 2004083432 (2004-09-01), None
patent: 2006000057 (2006-01-01), None
Aartsma-Rus et al. RNA 2007, 13: 1609-1624.
Adams et al. BMC Molecular Biology (2007) “Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries,” 8:57-64.
Alter et al. Nature Medicine (2006) “Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology,” 12:175-177.
McClorey et al. (2006) “Antisense oligonucleotide-induced exon skipping restores dystrophin expr ession in vitro in acanine model of DMD,” 13: 1375-1381.

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