Organic compounds -- part of the class 532-570 series – Organic compounds – Carbohydrates or derivatives
Reexamination Certificate
2005-12-05
2010-11-23
Bowman, Amy (Department: 1635)
Organic compounds -- part of the class 532-570 series
Organic compounds
Carbohydrates or derivatives
C536S023100, C536S024310, C536S024330, C514S04400A
Reexamination Certificate
active
07838657
ABSTRACT:
The present invention is directed to methods and compositions capable of blocking the inhibitory effect of a newly-identified intronic inhibitory sequence element, named ISS-N1 (for “intronic splicing silencer”), located in the SMN2 gene. The compositions and methods of the instant invention include oligonucleotide reagents (e.g., oligoribonucleotides) that effectively target the SMN2 ISS-N1 site in the SMN2 pre-mRNA, thereby modulating the splicing of SMN2 pre-mRNA to include exon 7 in the processed transcript. The ISS-N1 blocking agents of the invention cause elevated expression of SMN protein, thus compensating for the loss of SMN protein expression commonly observed in subjects with spinal muscular atrophy (SMA).
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International Search Report for Application No. PCT/US06/24469, dated Sep. 13, 2007.
Androphy Elliot J.
Singh Natalia N.
Singh Nirmal K.
Singh Ravindra N.
Bowman Amy
Lahive & Cockfield LLP
Milasincic Esq. Debra J.
University of Massachusetts
Williams Megan E.
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