Antisense Oligonucleotides for Treatment of Spinal Muscular Atrophy
OwnerArthur Burghes, Vicki McGovern +1inventors
InventorsArthur Burghes, Vicki McGovern, Thomas Prior
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Abstract
Various aspects of the present invention are directed to compounds targeted to various regions of the survival motor neuron 2 (SMN2) gene. Such compounds may be used to increase incorporation of exon 7 in processed transcripts of SMN2. Such compounds may therefore be useful in increasing the amount of full-length SMN protein produced by the SMN2 gene. As such, these compounds may provide a therapeutic approach for treatment of spinal muscular atrophy (SMA).
Claims20
An antisense oligonucleotide having a nucleotide sequence that is complementary to a portion of a sequence of intron 6, intron 7, or exon 7 of a survival motor neuron (SMN) 2 gene, the nucleotide sequence of the antisense oligonucleotide being chosen from [SEQ ID NO 1], [SEQ ID NO 2], [SEQ ID NO 4], [SEQ ID NO 5], [SEQ ID NO 6], [SEQ ID NO 7], [SEQ ID NO 8], [SEQ ID NO 9], [SEQ ID NO 10], [SEQ ID NO 11], [SEQ ID NO 12], [SEQ ID NO 13], [SEQ ID NO 14], [SEQ ID NO 15], [SEQ ID NO 16], and [SEQ ID NO 17].
A method of incorporating exon 7 of the SMN2 gene into transcripts of the SMN2 gene, the method comprising:
- administering to a subject an antisense oligonucleotide having a nucleotide sequence:
- (i) that is chosen from [SEQ ID NO 1], [SEQ ID NO 2], [SEQ ID NO 4], [SEQ ID NO 5], [SEQ ID NO 6], [SEQ ID NO 7], [SEQ ID NO 8], [SEQ ID NO 9], [SEQ ID NO 10], [SEQ ID NO 11], [SEQ ID NO 12], [SEQ ID NO 13], [SEQ ID NO 14], [SEQ ID NO 15], [SEQ ID NO 16], and [SEQ ID NO 17];
- or
- (ii) which is sufficiently homologous to [SEQ ID NO 1], [SEQ ID NO 2], [SEQ ID NO 4], [SEQ ID NO 5], [SEQ ID NO 6], [SEQ ID NO 7], [SEQ ID NO 8], [SEQ ID NO 9], [SEQ ID NO 10], [SEQ ID NO 11], [SEQ ID NO 12], [SEQ ID NO 13], [SEQ ID NO 14], [SEQ ID NO 15], [SEQ ID NO 16], or [SEQ ID NO 17];
- such that the nucleotide sequence of the antisense oligonucleotide is complementary to a portion of a sequence of intron 6, intron 7, or exon 7 of the SMN2 gene.
A composition for promoting the incorporation of exon 7 of the SMN2 gene into transcripts of the SMN2 gene, the composition comprising:
- at least a first antisense oligonucleotide having a nucleotide sequence:
- (i) that is chosen from [SEQ ID NO 1], [SEQ ID NO 2], [SEQ ID NO 4], [SEQ ID NO 5], [SEQ ID NO 6], [SEQ ID NO 7], [SEQ ID NO 8], [SEQ ID NO 9], [SEQ ID NO 10], [SEQ ID NO 11], [SEQ ID NO 12], [SEQ ID NO 13], [SEQ ID NO 14], [SEQ ID NO 15], [SEQ ID NO 16], and [SEQ ID NO 17];
- or
- (ii) which is sufficiently homologous to [SEQ ID NO 1], [SEQ ID NO 2], [SEQ ID NO 4], [SEQ ID NO 5], [SEQ ID NO 6], [SEQ ID NO 7], [SEQ ID NO 8], [SEQ ID NO 9], [SEQ ID NO 10], [SEQ ID NO 11], [SEQ ID NO 12], [SEQ ID NO 13], [SEQ ID NO 14], [SEQ ID NO 15], [SEQ ID NO 16], or [SEQ ID NO 17];
- such that the nucleotide sequence of the antisense oligonucleotide is complementary to a portion of a sequence of intron 6, intron 7, or exon 7 of the SMN2 gene.
Dependencies are inferred from the claim text.