IL203273A - Molecules for targeting compounds to muscle cells that contain conjugate, peptide or peptidomic and contain the lgaqsnf sequence associated with an active biological or diagnostic moiety. - Google Patents

Molecules for targeting compounds to muscle cells that contain conjugate, peptide or peptidomic and contain the lgaqsnf sequence associated with an active biological or diagnostic moiety.

Info

Publication number
IL203273A
IL203273A IL203273A IL20327310A IL203273A IL 203273 A IL203273 A IL 203273A IL 203273 A IL203273 A IL 203273A IL 20327310 A IL20327310 A IL 20327310A IL 203273 A IL203273 A IL 203273A
Authority
IL
Israel
Prior art keywords
peptide
moiety
muscle
group
conjugate
Prior art date
Application number
IL203273A
Other languages
English (en)
Hebrew (he)
Inventor
Johannes Antonius Heemskerk
Judith Christina Theodora Van Deutekom
Petra Van Kuik-Romeijn
Gerard Johannes Platenburg
Original Assignee
Prosensa Technologies Bv
Johannes Antonius Heemskerk
Judith Christina Theodora Van Deutekom
Petra Van Kuik-Romeijn
Gerard Johannes Platenburg
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 Prosensa Technologies Bv, Johannes Antonius Heemskerk, Judith Christina Theodora Van Deutekom, Petra Van Kuik-Romeijn, Gerard Johannes Platenburg filed Critical Prosensa Technologies Bv
Publication of IL203273A publication Critical patent/IL203273A/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/001—Preparation for luminescence or biological staining
    • A61K49/0013—Luminescence
    • A61K49/0017—Fluorescence in vivo
    • A61K49/005—Fluorescence in vivo characterised by the carrier molecule carrying the fluorescent agent
    • A61K49/0056—Peptides, proteins, polyamino acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/001—Preparation for luminescence or biological staining
    • A61K49/0013—Luminescence
    • A61K49/0017—Fluorescence in vivo
    • A61K49/0019—Fluorescence in vivo characterised by the fluorescent group, e.g. oligomeric, polymeric or dendritic molecules
    • A61K49/0021—Fluorescence in vivo characterised by the fluorescent group, e.g. oligomeric, polymeric or dendritic molecules the fluorescent group being a small organic molecule
    • A61K49/0032—Methine dyes, e.g. cyanine dyes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/001—Preparation for luminescence or biological staining
    • A61K49/0013—Luminescence
    • A61K49/0017—Fluorescence in vivo
    • A61K49/0019—Fluorescence in vivo characterised by the fluorescent group, e.g. oligomeric, polymeric or dendritic molecules
    • A61K49/0045—Fluorescence in vivo characterised by the fluorescent group, e.g. oligomeric, polymeric or dendritic molecules the fluorescent agent being a peptide or protein used for imaging or diagnosis in vivo
    • A61K49/0047—Green fluorescent protein [GFP]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Diabetes (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Endocrinology (AREA)
  • Pathology (AREA)
  • Radiology & Medical Imaging (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Emergency Medicine (AREA)
  • Child & Adolescent Psychology (AREA)
  • Pain & Pain Management (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
IL203273A 2007-07-12 2010-01-12 Molecules for targeting compounds to muscle cells that contain conjugate, peptide or peptidomic and contain the lgaqsnf sequence associated with an active biological or diagnostic moiety. IL203273A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP07112323 2007-07-12
PCT/NL2008/050475 WO2009008727A2 (en) 2007-07-12 2008-07-14 Molecules for targeting compounds to various selected organs or tissues

Publications (1)

Publication Number Publication Date
IL203273A true IL203273A (en) 2014-04-30

Family

ID=39471746

Family Applications (2)

Application Number Title Priority Date Filing Date
IL203273A IL203273A (en) 2007-07-12 2010-01-12 Molecules for targeting compounds to muscle cells that contain conjugate, peptide or peptidomic and contain the lgaqsnf sequence associated with an active biological or diagnostic moiety.
IL220979A IL220979A0 (en) 2007-07-12 2012-07-16 Molecules for targeting compounds to various selected organs or tissues

Family Applications After (1)

Application Number Title Priority Date Filing Date
IL220979A IL220979A0 (en) 2007-07-12 2012-07-16 Molecules for targeting compounds to various selected organs or tissues

Country Status (11)

Country Link
US (2) US8268962B2 (enExample)
EP (2) EP2505211B1 (enExample)
JP (1) JP5706157B2 (enExample)
CN (2) CN103212085A (enExample)
AU (1) AU2008273096B2 (enExample)
CA (2) CA2693048C (enExample)
CY (1) CY1117713T1 (enExample)
DK (1) DK2167136T3 (enExample)
IL (2) IL203273A (enExample)
NZ (1) NZ582521A (enExample)
WO (1) WO2009008727A2 (enExample)

Families Citing this family (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
JP5173838B2 (ja) 2006-02-13 2013-04-03 アレシア・バイオセラピューティクス・インコーポレーテッド 骨リモデリングのプロセスと関係があるポリヌクレオチドおよびポリペプチド配列
US8168181B2 (en) 2006-02-13 2012-05-01 Alethia Biotherapeutics, Inc. Methods of impairing osteoclast differentiation using antibodies that bind siglec-15
JP6047270B2 (ja) 2006-08-11 2016-12-21 バイオマリン テクノロジーズ ベー.フェー. Dnaリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段
NZ584793A (en) 2007-10-26 2012-05-25 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
CN102459595A (zh) 2009-04-24 2012-05-16 普罗森那技术公司 用于治疗dmd的包含肌苷的寡核苷酸
CN102048694B (zh) * 2009-11-06 2013-03-13 复旦大学 一种多肽修饰的肝肿瘤靶向纳米给药系统及其制备方法
AU2012246822B2 (en) * 2011-04-22 2017-05-25 Biomarin Technologies B.V. New compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (DM1)
WO2013103614A1 (en) * 2011-12-30 2013-07-11 Stc.Unm Crlf-2 binding peptides, protocells and viral-like particles useful in the treatment of cancer, including acute lymphoblastic leukemia (all)
CA2862628C (en) 2012-01-27 2021-08-24 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
PT2841578T (pt) 2012-04-23 2017-08-10 Biomarin Tech Bv Resumo
AU2013293062A1 (en) 2012-07-19 2014-07-24 Alethia Biotherapeutics Inc. Anti-Siglec-15 antibodies
CN103705465B (zh) * 2012-10-09 2016-01-13 复旦大学 一种微酸环境靶向多肽修饰的肿瘤靶向纳米给药系统及其制备方法
AU2015204452A1 (en) 2014-01-13 2016-08-11 Berg Llc Enolase 1 (Eno1) compositions and uses thereof
WO2016029125A1 (en) * 2014-08-22 2016-02-25 The Regents Of The University Of Michigan Peptide reagents and methods for detection and targeting of dysplasia, early cancer and cancer
CN104800858B (zh) * 2015-04-27 2017-11-21 中国医学科学院基础医学研究所 Hsp90抑制肽偶联物及其在肿瘤治疗中的应用
WO2016205397A2 (en) * 2015-06-15 2016-12-22 Klibanov Alexander L Target-specific delivery of therapeutic agents
WO2017011836A1 (en) * 2015-07-16 2017-01-19 Berg Llc Enolase 1 (eno1) compositions and uses thereof
WO2017120504A1 (en) 2016-01-08 2017-07-13 Durfee Paul N Osteotropic nanoparticles for prevention or treatment of bone metastases
WO2017136435A1 (en) 2016-02-01 2017-08-10 The Usa, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer National Institute Of Health Compounds for modulating fc-epsilon-ri-beta expression and uses thereof
BR112019009953A2 (pt) 2016-11-16 2019-10-08 Academisch Ziekenhuis Leiden substâncias direcionadas a vários órgãos ou tecidos selecionados
US11344629B2 (en) 2017-03-01 2022-05-31 Charles Jeffrey Brinker Active targeting of cells by monosized protocells
GB2563875B (en) * 2017-06-28 2020-08-19 Sutura Therapeutics Ltd Improvements in drug delivery
US12252708B2 (en) 2018-09-24 2025-03-18 Unm Rainforest Innovations Living mammalian cells modified with functional modular nanoparticles
US12331293B2 (en) 2018-11-02 2025-06-17 Biomarin Technologies B.V. Bispecific antisense oligonucleotides for dystrophin exon skipping
WO2020176716A1 (en) 2019-02-28 2020-09-03 Brinker C Jeffrey Modular metal–organic polyhedra superassembly compositions
EP4054639A4 (en) 2019-11-08 2025-04-30 North Carolina State University EXON SKIPPING OF FC-EPSILON-RI-BETA AND MS4A6A IN COMBINATION FOR THE TREATMENT OF ALLERGIC DISEASES
JP7727628B2 (ja) 2019-12-03 2025-08-21 アラマー バイオサイエンシーズ, インク. 核酸結合免疫サンドイッチアッセイ(nulisa)
US12704508B2 (en) 2021-06-02 2026-08-11 Alamar Biosciences, Inc. Nucleic acid linked immune-sandwich assay (NULISA)

Family Cites Families (104)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5034506A (en) * 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5541308A (en) * 1986-11-24 1996-07-30 Gen-Probe Incorporated Nucleic acid probes for detection and/or quantitation of non-viral organisms
US5766847A (en) * 1988-10-11 1998-06-16 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Process for analyzing length polymorphisms in DNA regions
DE3834636A1 (de) 1988-10-11 1990-04-19 Max Planck Gesellschaft Verfahren zur analyse von laengenpolymorphismen in dna-bereichen
US6867195B1 (en) * 1989-03-21 2005-03-15 Vical Incorporated Lipid-mediated polynucleotide administration to reduce likelihood of subject's becoming infected
US5608046A (en) * 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
FR2675803B1 (fr) * 1991-04-25 1996-09-06 Genset Sa Oligonucleotides fermes, antisens et sens et leurs applications.
AU659482B2 (en) 1991-06-28 1995-05-18 Massachusetts Institute Of Technology Localized oligonucleotide therapy
CA2082411A1 (en) 1991-06-28 1992-12-29 Robert D. Rosenberg Localized oligonucleotide therapy
US6200747B1 (en) * 1992-01-28 2001-03-13 North Shore University Hospital Research Corp. Method and kits for detection of fragile X specific, GC-rich DNA sequences
US5869252A (en) * 1992-03-31 1999-02-09 Abbott Laboratories Method of multiplex ligase chain reaction
US5418139A (en) * 1993-02-10 1995-05-23 University Of Iowa Research Foundation Method for screening for cardiomyopathy
CA2116280A1 (en) 1993-03-05 1994-09-06 Marcy E. Macdonald Huntingtin dna, protein and uses thereof
US5741645A (en) * 1993-06-29 1998-04-21 Regents Of The University Of Minnesota Gene sequence for spinocerebellar ataxia type 1 and method for diagnosis
US5627263A (en) * 1993-11-24 1997-05-06 La Jolla Cancer Research Foundation Integrin-binding peptides
DE4342605A1 (de) 1993-12-14 1995-06-22 Buna Gmbh Funktionalisierte Olefinhomo- und -copolymere
US5962332A (en) * 1994-03-17 1999-10-05 University Of Massachusetts Detection of trinucleotide repeats by in situ hybridization
AU2360195A (en) 1994-05-05 1995-11-29 Beckman Instruments, Inc. Oligonucleotide repeat arrays
US5968909A (en) * 1995-08-04 1999-10-19 Hybridon, Inc. Method of modulating gene expression with reduced immunostimulatory response
US5854223A (en) 1995-10-06 1998-12-29 The Trustees Of Columbia University In The City Of New York S-DC28 as an antirestenosis agent after balloon injury
US6300060B1 (en) * 1995-11-09 2001-10-09 Dana-Farber Cancer Institute, Inc. Method for predicting the risk of prostate cancer morbidity and mortality
DK0882061T3 (da) 1996-02-14 2004-09-27 Isis Pharmaceuticals Inc Sukkermodificerede gapped oligonukleotider
US6251589B1 (en) * 1996-07-18 2001-06-26 Srl, Inc. Method for diagnosing spinocerebellar ataxia type 2 and primers therefor
WO1998018920A1 (fr) 1996-10-30 1998-05-07 Srl, Inc. Fragments d'adnc du gene responsable de l'ataxie spino-cerebelleuse de type 2
US5853995A (en) 1997-01-07 1998-12-29 Research Development Foundation Large scale genotyping of diseases and a diagnostic test for spinocerebellar ataxia type 6
US20020137890A1 (en) * 1997-03-31 2002-09-26 Genentech, Inc. Secreted and transmembrane polypeptides and nucleic acids encoding the same
WO1998049345A1 (en) 1997-04-29 1998-11-05 Trustees Of Boston University Methods and compositions for targeted dna differential display
US6329501B1 (en) * 1997-05-29 2001-12-11 Auburn University Methods and compositions for targeting compounds to muscle
US6514755B1 (en) * 1998-08-18 2003-02-04 Regents Of The University Of Minnesota SCA7 gene and methods of use
US6280938B1 (en) * 1997-08-19 2001-08-28 Regents Of The University Of Minnesota SCA7 gene and method of use
US6794499B2 (en) * 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6130207A (en) * 1997-11-05 2000-10-10 South Alabama Medical Science Foundation Cell-specific molecule and method for importing DNA into a nucleus
JP3012923B2 (ja) * 1998-01-26 2000-02-28 新潟大学長 Cagリピート病の治療薬
KR100280219B1 (ko) * 1998-02-26 2001-04-02 이수빈 삼핵산 반복 서열을 이용한 신경정신 질환의 진단 방법 및 진단 시약
US6322978B1 (en) * 1998-04-20 2001-11-27 Joslin Diabetes Center, Inc. Repeat polymorphism in the frataxin gene and uses therefore
US6924355B2 (en) * 1998-09-01 2005-08-02 Genentech, Inc. PRO1343 polypeptides
WO2000018895A1 (en) * 1998-09-25 2000-04-06 The Children's Medical Center Corporation Short peptides which selectively modulate the activity of protein kinases
US6172216B1 (en) * 1998-10-07 2001-01-09 Isis Pharmaceuticals Inc. Antisense modulation of BCL-X expression
US6399575B1 (en) * 1998-11-10 2002-06-04 Auburn University Methods and compositions for targeting compounds to the central nervous system
US6133031A (en) * 1999-08-19 2000-10-17 Isis Pharmaceuticals Inc. Antisense inhibition of focal adhesion kinase expression
US20040226056A1 (en) * 1998-12-22 2004-11-11 Myriad Genetics, Incorporated Compositions and methods for treating neurological disorders and diseases
US20020049173A1 (en) * 1999-03-26 2002-04-25 Bennett C. Frank Alteration of cellular behavior by antisense modulation of mRNA processing
US6379698B1 (en) * 1999-04-06 2002-04-30 Isis Pharmaceuticals, Inc. Fusogenic lipids and vesicles
JP2000325085A (ja) * 1999-05-21 2000-11-28 Masafumi Matsuo デュシェンヌ型筋ジストロフィー治療剤
US20030236214A1 (en) * 1999-06-09 2003-12-25 Wolff Jon A. Charge reversal of polyion complexes and treatment of peripheral occlusive disease
WO2000078813A2 (en) * 1999-06-18 2000-12-28 Emory University Huntington disease cellular model: stably transfected pc12 cells expressing mutant huntingtin
EP1133993A1 (en) 2000-03-10 2001-09-19 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Substances for the treatment of spinal muscular atrophy
US20020187931A1 (en) 2000-04-13 2002-12-12 Michael Hayden Modulating cell survival by modulating huntingtin function
US6653467B1 (en) * 2000-04-26 2003-11-25 Jcr Pharmaceutical Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
WO2001083695A2 (en) 2000-04-28 2001-11-08 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof
WO2001085751A1 (en) * 2000-05-09 2001-11-15 Reliable Biopharmaceutical, Inc. Polymeric compounds useful as prodrugs
EP1294866B1 (en) * 2000-05-31 2010-04-14 Serono Genetics Institute S.A. Use of acrp30 globular head to promote increases in muscle mass and muscle differentiation
US20030124523A1 (en) * 2000-06-22 2003-07-03 Asselbergs Fredericus Alphonsus Maria Organic compounds
US6794192B2 (en) * 2000-06-29 2004-09-21 Pfizer Inc. Target
RU2165149C1 (ru) 2000-07-03 2001-04-20 Шапошников Валерий Геннадьевич Система формования и упаковки изделий из сахарной ваты
US6727355B2 (en) 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
EP1191097A1 (en) * 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
US6623927B1 (en) * 2000-11-08 2003-09-23 Council Of Scientific And Industrial Research Method of detection of allelic variants of SCA2 gene
AU2002236499A1 (en) * 2000-11-30 2002-06-11 Uab Research Foundation Receptor-mediated uptake of peptides that bind the human transferrin receptor
TWI329129B (en) * 2001-02-08 2010-08-21 Wyeth Corp Modified and stabilized gdf propeptides and uses thereof
US6902896B2 (en) * 2001-05-11 2005-06-07 Regents Of The University Of Minnesota Intron associated with myotonic dystrophy type 2 and methods of use
EP1627061B1 (en) 2001-05-18 2009-08-12 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA)
US20050277133A1 (en) * 2001-05-18 2005-12-15 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
EP1423537A4 (en) * 2001-08-07 2006-11-29 Univ Delaware COMPOSITIONS AND METHODS FOR PREVENTING AND TREATING HUNTINGTON'S DISEASE
US20060074034A1 (en) * 2001-09-17 2006-04-06 Collins Douglas A Cobalamin mediated delivery of nucleic acids, analogs and derivatives thereof
KR20030035047A (ko) 2001-10-29 2003-05-09 (주)바이오코돈 Bmp-4 유전자 발현을 이용한 편평태선 질환의 치료 및진단방법
WO2003037172A2 (en) * 2001-11-01 2003-05-08 Gpc Biotech Inc. Endothelial-cell binding peptides for diagnosis and therapy
US20030134790A1 (en) * 2002-01-11 2003-07-17 University Of Medicine And Dentistry Of New Jersey Bone Morphogenetic Protein-2 And Bone Morphogenetic Protein-4 In The Treatment And Diagnosis Of Cancer
WO2003069330A1 (en) * 2002-02-11 2003-08-21 The Trustees Of Columbia University In The City Of New York System and method for identifying proteins involved in force-initiated signal transduction
US20050096284A1 (en) * 2002-02-20 2005-05-05 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
US7771727B2 (en) 2002-03-01 2010-08-10 The Administrators Of The Tulane Educational Fund Conjugates of therapeutic or cytotoxic agents and biologically active peptides
US20040101852A1 (en) * 2002-11-21 2004-05-27 Isis Pharmaceuticals Inc. Modulation of CGG triplet repeat binding protein 1 expression
ITRM20020253A1 (it) 2002-05-08 2003-11-10 Univ Roma Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.
EP1380644A1 (en) 2002-07-08 2004-01-14 Kylix B.V. The use of specified TCF target genes to identify drugs for the treatment of cancer, in particular colorectal cancer, in which TCF/beta-catenin/WNT signalling plays a central role
US7589059B2 (en) 2002-07-26 2009-09-15 Roche Madison Inc. Delivery of molecules and complexes to mammalian cells in vivo
US20050255086A1 (en) 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
JP2005535318A (ja) 2002-08-12 2005-11-24 ユニヴェルシテ ドゥ シェルブルック プレメッセンジャーrnaのスプライス部位選択の再プログラミング方法
GB0219143D0 (en) 2002-08-16 2002-09-25 Univ Leicester Modified tailed oligonucleotides
ES2283608T3 (es) * 2002-10-23 2007-11-01 Centre For Research And Technology Hellas/Institute Of Agrobiotechnology In.A Secuencias peptidicas de union a la proteina prionica.
US7892793B2 (en) 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
EP2305813A3 (en) * 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
EP2530155B1 (en) 2002-11-25 2016-03-16 Masafumi Matsuo ENA nucleic acid drugs modifying splicing in mRNA precursor
GB0228079D0 (en) 2002-12-02 2003-01-08 Laxdale Ltd Huntington's Disease
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
AU2004239114B2 (en) 2003-05-14 2008-03-13 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
WO2004108157A2 (en) 2003-06-02 2004-12-16 Wyeth Use of myostatin (gdf8) inhibitors in conjunction with corticosteroids for treating neuromuscular disorders
ES2302898T3 (es) 2003-07-11 2008-08-01 Lbr Medbiotech B.V. Transferencia de genes a celulas musculares mediada por el receptor de manosa-6-fosfato.
US20050054752A1 (en) * 2003-09-08 2005-03-10 O'brien John P. Peptide-based diblock and triblock dispersants and diblock polymers
US7355018B2 (en) * 2003-09-30 2008-04-08 Regeneron Pharmaceuticals, Inc. Modified IGF1 polypeptides with increased stability and potency
EP1680439A2 (en) * 2003-10-14 2006-07-19 Kernel Biopharma Inc. Dual phase-pna conjugates for the delivery of pna through the blood brain barrier
US20050191636A1 (en) 2004-03-01 2005-09-01 Biocept, Inc. Detection of STRP, such as fragile X syndrome
WO2005105995A2 (en) 2004-04-14 2005-11-10 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA)
CA2566286A1 (en) 2004-05-11 2005-12-08 Rnai Co., Ltd. Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same
ES2627917T3 (es) 2004-06-28 2017-08-01 The University Of Western Australia Oligonucleótidos no codificantes para la inducción de la omisión exónica y métodos de uso de los mismos
EP1618881A1 (en) 2004-07-20 2006-01-25 Santhera Pharmaceuticals (Schweiz) GmbH Use of non-glucocorticoid steroids for the treatment of muscular dystrophy
ITRM20040568A1 (it) 2004-11-18 2005-02-18 Uni Degli Studi Di Roma Tor Vergata Uso della tecnica "phage display" per l'identificazione di peptidi con capacita' di legame a cellule staminali/progenitore, peptidi cosi' ottenuti e loro usi.
US20060148740A1 (en) * 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
CA2596588C (en) 2005-01-31 2017-06-27 University Of Iowa Research Foundation Nucleic acid silencing of huntington's disease gene
US20060257912A1 (en) 2005-05-06 2006-11-16 Medtronic, Inc. Methods and sequences to suppress primate huntington gene expression
CN100393320C (zh) * 2005-06-24 2008-06-11 奥林格斯技术有限公司 治疗肌肉萎缩的寡核苷酸药物
ATE522626T1 (de) 2005-06-28 2011-09-15 Medtronic Inc Verfahren und nukleotid-sequenzen, die bevorzugt die expression eines mutierten huntingtin-genes unterdrücken
US7906617B2 (en) * 2005-12-15 2011-03-15 E. I. Du Pont De Nemours And Company Polyethylene binding peptides and methods of use
US20100248239A1 (en) * 2009-03-24 2010-09-30 Mayo Foundation For Medical Education And Research Methods and materials for detecting fragile x mutations
US20110166081A1 (en) * 2009-12-03 2011-07-07 University Of Iowa Research Foundation Alpha-dystroglycan as a Protein Therapeutic

Also Published As

Publication number Publication date
IL220979A0 (en) 2012-08-30
CN103212085A (zh) 2013-07-24
CA2942716A1 (en) 2009-01-15
HK1145635A1 (en) 2011-04-29
CA2693048C (en) 2016-10-18
EP2505211A1 (en) 2012-10-03
US8268962B2 (en) 2012-09-18
NZ582521A (en) 2011-09-30
EP2167136B1 (en) 2016-04-20
US8519097B2 (en) 2013-08-27
CN101815535A (zh) 2010-08-25
EP2167136A2 (en) 2010-03-31
CN101815535B (zh) 2013-04-24
US20100184948A1 (en) 2010-07-22
AU2008273096B2 (en) 2013-05-02
AU2008273096A1 (en) 2009-01-15
CY1117713T1 (el) 2017-05-17
US20120322724A1 (en) 2012-12-20
CA2693048A1 (en) 2009-01-15
CA2942716C (en) 2019-07-09
JP5706157B2 (ja) 2015-04-22
WO2009008727A2 (en) 2009-01-15
EP2505211B1 (en) 2020-04-08
JP2010533171A (ja) 2010-10-21
WO2009008727A3 (en) 2009-08-13
DK2167136T3 (en) 2016-07-25

Similar Documents

Publication Publication Date Title
CA2942716C (en) Molecules for targeting compounds to various selected organs or tissues
US8609065B2 (en) Molecules for targeting compounds to various selected organs, tissues or tumor cells
KR102646799B1 (ko) 다양하게 선택된 기관 또는 조직을 표적화하기 위한 물질
ES2577712T3 (es) Moléculas para direccionar compuestos a varios órganos o tejidos seleccionados
HK1176286A (en) Molecules for targeting compounds to various selected organs or tissues
WO2024041372A1 (zh) 一种用于有效递送核酸的分支链结构多肽载体及其变化形式
HK1145635B (en) Molecules for targeting compounds to various selected organs or tissues
HK40015212B (en) Substances for targeting various selected organs or tissues

Legal Events

Date Code Title Description
FF Patent granted
KB Patent renewed
KB Patent renewed