BENNETT C. FRANK,HUNG GENE,RIGO FRANK,KRAINER ADRIAN R.,HUA YIMIN,PASSINI MARCO A.,SHIHABUDDIN LAMYA,CHENG SENG H.,KLINGER KATHERINE W.
申请号：
NZ59707110
公开号：
NZ597071A
申请日:
2010.06.17
申请国别(地区):
NZ
年份:
2014
代理人:
摘要：
Disclosed is the use of an antisense compound comprising an antisense oligonucleotide complementary to intron 7 of a nucleic acid encoding human SMN2 pre-mRNA, wherein each nucleoside of the antisense oligonucleotide comprises a modified sugar moiety, wherein each modified sugar moiety is a 2&rsquo-methoxyethyl sugar moiety, in the manufacture of a medicament for use in treating a disease or condition associated with survival motor neuron 1 (SMN1) in a subject, wherein the medicament is formulated for administration into the cerebrospinal fluid. Further disclosed is the use of an antisense compound comprising an antisense oligonucleotide complementary to intron 7 of a nucleic acid encoding human SMN2, wherein each nucleoside of the antisense oligonucleotide comprises a modified sugar moiety, wherein each modified sugar moiety is a 2&rsquo-methoxyethyl sugar moiety, in the manufacture of a medicament for treating a disease or condition associated with survival motor neuron 1 (SMN1) in a subject, wherein the medicament is formulated for increasing inclusion of exon 7 of SMN2 mRNA or amino acids in a SMN2 polypeptide in a motoneuron in a subject.
