WO2004113867A3 - Exon analysis - Google Patents

Exon analysis Download PDF

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Publication number
WO2004113867A3
WO2004113867A3 PCT/US2004/019081 US2004019081W WO2004113867A3 WO 2004113867 A3 WO2004113867 A3 WO 2004113867A3 US 2004019081 W US2004019081 W US 2004019081W WO 2004113867 A3 WO2004113867 A3 WO 2004113867A3
Authority
WO
WIPO (PCT)
Prior art keywords
determining
present
novel
splicing
exonic splicing
Prior art date
Application number
PCT/US2004/019081
Other languages
French (fr)
Other versions
WO2004113867A2 (en
Inventor
Ravindra N Singh
Natalia N Singh
Elliot J Androphy
Original Assignee
Univ Massachusetts
Ravindra N Singh
Natalia N Singh
Elliot J Androphy
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Massachusetts, Ravindra N Singh, Natalia N Singh, Elliot J Androphy filed Critical Univ Massachusetts
Publication of WO2004113867A2 publication Critical patent/WO2004113867A2/en
Publication of WO2004113867A3 publication Critical patent/WO2004113867A3/en

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1051—Gene trapping, e.g. exon-, intron-, IRES-, signal sequence-trap cloning, trap vectors

Abstract

The present invention provides methods of analyzing an entire exon or a portion thereof using a novel cell-based SELEX procedure. Described is a novel and accurate means of determining the location of exonic splicing enhancers (ESEs) and exonic splicing silencers (ESSs). The methods described have wide application, e.g., for determining the molecular etiology of human diseases associated with splicing errors, and provides new therapeutic targets for such diseases. Nucleotide sequences (aptamers) identified by the present invention are also provided.
PCT/US2004/019081 2003-06-16 2004-06-16 Exon analysis WO2004113867A2 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US47846903P 2003-06-16 2003-06-16
US60/478,469 2003-06-16
US57638004P 2004-06-01 2004-06-01
US60/576,380 2004-06-01

Publications (2)

Publication Number Publication Date
WO2004113867A2 WO2004113867A2 (en) 2004-12-29
WO2004113867A3 true WO2004113867A3 (en) 2006-02-02

Family

ID=33544372

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2004/019081 WO2004113867A2 (en) 2003-06-16 2004-06-16 Exon analysis

Country Status (1)

Country Link
WO (1) WO2004113867A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9328346B2 (en) 2010-11-12 2016-05-03 The General Hospital Corporation Polycomb-associated non-coding RNAs

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK1910395T3 (en) 2005-06-23 2013-02-04 Isis Pharmaceuticals Inc Compositions and Methods for Modulating SMN2 Splicing
WO2009151546A2 (en) * 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
US8986935B2 (en) 2008-08-13 2015-03-24 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
EP3305302B1 (en) 2009-06-17 2018-09-19 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
AP2014008100A0 (en) 2012-05-16 2014-12-31 Gen Hospital Corp Compositions and methods for modulating hemoglobingene family expression
WO2013173608A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating mecp2 expression
US10174323B2 (en) 2012-05-16 2019-01-08 The General Hospital Corporation Compositions and methods for modulating ATP2A2 expression
AU2013262656A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating UTRN expression
JP2015523854A (en) 2012-05-16 2015-08-20 ラナ セラピューティクス インコーポレイテッド Compositions and methods for modulating SMN gene family expression
EP2943225A4 (en) 2013-01-09 2016-07-13 Ionis Pharmaceuticals Inc Compositions and methods for modulation of smn2 splicing in a subject
EP3797780B1 (en) 2014-04-17 2022-09-14 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
BR112017004056A2 (en) 2014-09-12 2017-12-05 Biogen Ma Inc compositions and methods for detecting smn protein in an individual and treating an individual
CA2966044A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
WO2016149455A2 (en) 2015-03-17 2016-09-22 The General Hospital Corporation The rna interactome of polycomb repressive complex 1 (prc1)
WO2017218884A1 (en) 2016-06-16 2017-12-21 Ionis Pharmaceuticals, Inc. Combinations for the modulation of smn expression
KR20220148230A (en) 2020-02-28 2022-11-04 아이오니스 파마수티컬즈, 인코포레이티드 Compounds and methods for modulating SMN2

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
COULTER ET AL: "Identification of a New Class of Exonic Splicing Enhancers by In Vivo Selection", MOLECULAR AND CELLULAR BIOLOGY, vol. 17, April 1997 (1997-04-01), pages 2143 - 2150, XP002993118 *
TIAN ET AL: "Strong RNA Splicing Enhancers Identified by a Modified Method of Cycled Selection Interact with SR Protein", J. BIOL. CHEM., vol. 276, no. 36, 7 September 2001 (2001-09-07), pages 33833 - 33839, XP002993119 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9328346B2 (en) 2010-11-12 2016-05-03 The General Hospital Corporation Polycomb-associated non-coding RNAs

Also Published As

Publication number Publication date
WO2004113867A2 (en) 2004-12-29

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