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.