发明名称 Compositions and methods for modulating MECP2 expression
摘要 <p>Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of MECP2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of MECP2. Methods for modulating expression of MECP2 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of MECP2.</p>
申请公布号 AU2013262709(A1) 申请公布日期 2015.01.22
申请号 AU20130262709 申请日期 2013.05.16
申请人 RANA THERAPEUTICS, INC.;THE GENERAL HOSPITAL CORPORATION D/B/A MASSACHUSETTS GENERAL HOSPITAL 发明人 KRIEG, ARTHUR M.;SUBRAMANIAN, ROMESH;MCSWIGGEN, JAMES;LEE, JEANNIE T.
分类号 C12N15/11;A61K31/7088;A61P25/00;C07H21/00 主分类号 C12N15/11
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