WO2001036627A3 - Pharmaceutical compositions comprising acetylcholinesterase antisense deoxynucleotides for the treatment of muscular and neuromuscular disorders - Google Patents

Pharmaceutical compositions comprising acetylcholinesterase antisense deoxynucleotides for the treatment of muscular and neuromuscular disorders Download PDF

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Publication number
WO2001036627A3
WO2001036627A3 PCT/IL2000/000763 IL0000763W WO0136627A3 WO 2001036627 A3 WO2001036627 A3 WO 2001036627A3 IL 0000763 W IL0000763 W IL 0000763W WO 0136627 A3 WO0136627 A3 WO 0136627A3
Authority
WO
WIPO (PCT)
Prior art keywords
treatment
acetylcholinesterase
muscular
pharmaceutical compositions
neuromuscular disorders
Prior art date
Application number
PCT/IL2000/000763
Other languages
French (fr)
Other versions
WO2001036627A2 (en
Inventor
Hermona Soreq
Shlomo Seidman
Original Assignee
Yissum Res Dev Co
Hermona Soreq
Shlomo Seidman
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 Yissum Res Dev Co, Hermona Soreq, Shlomo Seidman filed Critical Yissum Res Dev Co
Priority to AU15461/01A priority Critical patent/AU1546101A/en
Publication of WO2001036627A2 publication Critical patent/WO2001036627A2/en
Publication of WO2001036627A3 publication Critical patent/WO2001036627A3/en

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    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes

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  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The present invention relates to a pharmaceutical composition for the treatment and/or prevention of a progressive neuromuscular disorder, and for use in decreasing muscle fatigue, comprising low concentrations of a synthetic antisense oligonucleotide targeted to the common coding domain of acetylcholine esterase (AChE) mRNA. The present invention further relates to a novel method for the diagnosis of progressive neuromuscular disorders.
PCT/IL2000/000763 1999-11-16 2000-11-16 Pharmaceutical compositions comprising acetylcholinesterase antisense deoxynucleotides for the treatment of muscular and neuromuscular disorders WO2001036627A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU15461/01A AU1546101A (en) 1999-11-16 2000-11-16 Pharmaceutical compositions comprising acetylcholinesterase antisense deoxynucleotides for the treatment of muscular and neuromuscular disorders

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
IL132972 1999-11-16
IL13297299A IL132972A0 (en) 1999-11-16 1999-11-16 Pharmaceutical compositions comprising acetylcholinesterase antisense deoxynucleotides for the treatment of muscular and neuromuscular disorders

Publications (2)

Publication Number Publication Date
WO2001036627A2 WO2001036627A2 (en) 2001-05-25
WO2001036627A3 true WO2001036627A3 (en) 2001-10-04

Family

ID=11073493

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IL2000/000763 WO2001036627A2 (en) 1999-11-16 2000-11-16 Pharmaceutical compositions comprising acetylcholinesterase antisense deoxynucleotides for the treatment of muscular and neuromuscular disorders

Country Status (3)

Country Link
AU (1) AU1546101A (en)
IL (1) IL132972A0 (en)
WO (1) WO2001036627A2 (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 (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL143379A (en) 2001-05-24 2013-11-28 Yissum Res Dev Co Antisense oligonucleotide against the r isophorm of human ache and uses thereof
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
CN104583401A (en) 2012-05-16 2015-04-29 Rana医疗有限公司 Compositions and methods for modulating ATP2A2 expression
KR20160074368A (en) 2012-05-16 2016-06-28 라나 테라퓨틱스, 인크. Compositions and methods for modulating utrn expression
DK2850186T3 (en) 2012-05-16 2019-04-08 Translate Bio Ma Inc COMPOSITIONS AND PROCEDURES FOR MODULATING SMN GENFAMILY EXPRESSION
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
US10174315B2 (en) 2012-05-16 2019-01-08 The General Hospital Corporation Compositions and methods for modulating hemoglobin gene family expression
WO2016070060A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
US10900036B2 (en) 2015-03-17 2021-01-26 The General Hospital Corporation RNA interactome of polycomb repressive complex 1 (PRC1)
US20220242931A1 (en) * 2019-05-13 2022-08-04 The Trustees Of The University Of Pennsylvania Compositions and methods of acetylcholine receptor chimeric autoantibody receptor cells

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993021202A1 (en) * 1992-04-15 1993-10-28 Yissum Research Development Company Of The Hebrew University Of Jerusalem Synthetic antisense oligodeoxynucleotides and pharmaceutical compositions containing them
WO1998026062A2 (en) * 1996-12-12 1998-06-18 Yissum Research Development Company Of The Hebrew University Of Jerusalem Synthetic antisense oligodeoxynucleotides and pharmaceutical compositions containing them
US5932780A (en) * 1994-02-28 1999-08-03 Yissum Research Development Company Of Hebrew University Of Jerusalem Transgenic non-human animal assay system for anti-cholinesterase substances

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993021202A1 (en) * 1992-04-15 1993-10-28 Yissum Research Development Company Of The Hebrew University Of Jerusalem Synthetic antisense oligodeoxynucleotides and pharmaceutical compositions containing them
US5932780A (en) * 1994-02-28 1999-08-03 Yissum Research Development Company Of Hebrew University Of Jerusalem Transgenic non-human animal assay system for anti-cholinesterase substances
WO1998026062A2 (en) * 1996-12-12 1998-06-18 Yissum Research Development Company Of The Hebrew University Of Jerusalem Synthetic antisense oligodeoxynucleotides and pharmaceutical compositions containing them

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
SEIDMAN S. ET AL.: "Antisense technologies have a future fighting neurodegenerative diseases", ANTISENSE & NUCLEIC ACID DRUG DEVELOPMENT, vol. 9, August 1999 (1999-08-01), pages 333 - 340, XP000984531 *
TALMA BRENNER ET AL: "Antisense approach to the anticholinesterase management of myasthenia gravis.", JOURNAL OF AUTOIMMUNITY, no. SUPPL., 1999, 2nd International Congress on Autoimmunity;Tel Aviv, Israel; March 7-11, 1999, pages 22, XP000986370, ISSN: 0896-8411 *

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
IL132972A0 (en) 2001-03-19
AU1546101A (en) 2001-05-30
WO2001036627A2 (en) 2001-05-25

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