Compositions and methods for modulating mecp2 expression

a composition and mecp2 technology, applied in the field of oligonucleotide based compositions, can solve the problems of slowed brain and head growth, seizures, ambulatory problems, etc., and achieve the effect of relieving or enhancing the expression of mecp2 and preventing the mediated repression of mecp2

Inactive Publication Date: 2015-06-04
RANA THERAPEUTICS INC +1
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Benefits of technology

[0005]Aspects of the invention disclosed herein provide methods and compositions that are useful for upregulating MECP2 in cells. In some embodiments, single stranded oligonucleotides are provided that target a PRC2-associated region of a MECP2 gene (e.g., human MECP2) and thereby cause upregulation...

Problems solved by technology

Rett syndrome is a developmental disorder of the brain occurring mostly in females characterized by normal early development, followed by a slowing of development resulting in loss of control of the hands, loss of speec...

Method used

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  • Compositions and methods for modulating mecp2 expression
  • Compositions and methods for modulating mecp2 expression
  • Compositions and methods for modulating mecp2 expression

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examples

[0193]The invention is further described in the following examples, which do not limit the scope of the invention described in the claims.

Materials and Methods:

Real Time PCR

[0194]RNA is harvested from the cells using Promega SV 96 Total RNA Isolation system or Trizol omitting the DNAse step. RNA harvested from cells is normalized so that equal RNA is input to each reverse transcription reaction. Reverse transcriptase reactions are performed using the Superscript II kit and real time PCR performed on cDNA samples using icycler SYBR green chemistry (Biorad). A baseline level of mRNA expression for MECP2 is determined through quantitative PCR as outlined above. Baseline levels are also determined for mRNA of various housekeeping genes which are constitutively expressed. A “control” housekeeping gene with approximately the same level of baseline expression as the target gene is chosen for comparison purposes.

Cell Culture

[0195]Human hepatocyte Hep3B, human hepatocyte HepG2 cells, mouse h...

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Abstract

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.

Description

CROSS-REFERENCE TO RELATED APPLICATIONS[0001]This application claims the benefit under 35 U.S.C. §119(e) of U.S. Provisional Application No. 61 / 648,051, entitled “COMPOSITIONS AND METHODS FOR MODULATING MECP2 EXPRESSION”, filed May 16, 2012, which is incorporated herein by reference in its entirety.FIELD OF THE INVENTION[0002]The invention relates to oligonucleotide based compositions, as well as methods of using oligonucleotide based compositions for treating disease.BACKGROUND OF THE INVENTION[0003]Rett syndrome is a developmental disorder of the brain occurring mostly in females characterized by normal early development, followed by a slowing of development resulting in loss of control of the hands, loss of speech, breathing problems, slowed brain and head growth, ambulatory problems, seizures, and mental retardation. Rett syndrome affects approximately 1 in 10,000 live female births. Most cases of Rett syndrome are caused by a mutation in the methyl CpG binding protein 2, or MEC...

Claims

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Application Information

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IPC IPC(8): C12N15/113A61K31/7115A61K31/712A61K47/48A61K31/7125
CPCC12N15/113A61K47/48246A61K31/7125A61K31/712A61K31/7115A61K47/4813C12N2310/3231C12N2310/315C07H21/00C07H21/02C07H21/04C12N2310/11C12N2310/321A61K47/555A61K47/64A61P25/00A61P43/00C12N2310/3521A61K47/61A61K47/6455
Inventor KRIEG, ARTHUR M.SUBRAMANIAN, ROMESHMCSWIGGEN, JAMESLEE, JEANNIE T.
Owner RANA THERAPEUTICS INC
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