公开(公告)号：US20210102203A1

公开(公告)日：2021-04-08

申请号：US16876271

申请日：2020-05-18

申请人： Translate Bio MA, Inc. ,  The General Hospital Corporation d/b/a Massachusetts General Hospital

发明人： Arthur M. Krieg ,  Romesh R. Subramanian ,  James McSwiggen ,  Jeannie T. Lee

IPC分类号： C12N15/113 ,  A61K47/61 ,  A61K47/64 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  C07H21/00 ,  C07H21/02 ,  C07H21/04 ,  A61K47/54

摘要： 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.

公开(公告)号：US11788089B2

公开(公告)日：2023-10-17

申请号：US16876271

申请日：2020-05-18

申请人： The General Hospital Corporation

发明人： Arthur M. Krieg ,  Romesh R. Subramanian ,  James McSwiggen ,  Jeannie T. Lee

IPC分类号： C07H21/02 ,  C07H21/04 ,  C12N15/113 ,  A61K47/61 ,  A61K47/64 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  C07H21/00 ,  A61K47/54

CPC分类号： C12N15/113 ,  A61K31/712 ,  A61K31/7115 ,  A61K31/7125 ,  A61K47/555 ,  A61K47/61 ,  A61K47/64 ,  A61K47/6455 ,  C07H21/00 ,  C07H21/02 ,  C07H21/04 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/321 ,  C12N2310/3521

摘要： 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.