发明名称 Treatment of membrane bound transcription factor peptidase, site 1 (MBTPS1) related diseases by inhibition of natural antisense transcript to MBTPS1
摘要 The present invention relates to antisense oligonuclotides that modulate the expression of and/or function of Membrane Bound Transcription Factor Peptidase, site 1 (MBTPS1), in particular, by targeting natural antisense polynucleotides of Membrane Bound Transcription Factor Peptidase, site 1 (MBTPS1). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of MBTPS1.
申请公布号 US9173895(B2) 申请公布日期 2015.11.03
申请号 US201013516097 申请日期 2010.12.15
申请人 CuRNA, Inc. 发明人 Collard Joseph;Khorkova Sherman Olga
分类号 C12N15/113;A61K31/7088 主分类号 C12N15/113
代理机构 CuRNA, Inc. 代理人 CuRNA, Inc. ;Browder Monte R.
主权项 1. A method of upregulating a function of and/or the expression of a Membrane Bound Transcription Factor Peptidase, site 1 (MBTPS1) poly in patient cells or tissues in vivo or in vitro comprising: contacting said cells or tissues with at least one antisense oligonucleotide 15 to 30 nucleotides in length wherein said at least one oligonucleotide is specific for and specifically hybridizes to a natural antisense polynucleotide of the MBTPS1 gene and has at least 80% sequence identity to a reverse complement of a polynucleotide comprising 15 to 30 nucleotides within nucleotides 1 to 1240 of SEQ ID NO: 2; thereby upregulating a function of and/or the expression of the Membrane Bound Transcription Factor Peptidase, site 1 (MBTPS1) polynucleotide in patient cells or tissues in vivo or in vitro.
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