WO2011000516A3 - Gamma globin therapy - Google Patents

Gamma globin therapy Download PDF

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Publication number
WO2011000516A3
WO2011000516A3 PCT/EP2010/003854 EP2010003854W WO2011000516A3 WO 2011000516 A3 WO2011000516 A3 WO 2011000516A3 EP 2010003854 W EP2010003854 W EP 2010003854W WO 2011000516 A3 WO2011000516 A3 WO 2011000516A3
Authority
WO
WIPO (PCT)
Prior art keywords
therapy
gamma globin
gene expression
globin
globin gene
Prior art date
Application number
PCT/EP2010/003854
Other languages
French (fr)
Other versions
WO2011000516A2 (en
Inventor
Franklin Gerardus Grosveld
Farzin Pourfarzad
Original Assignee
Erasmus University Medical Center Rotterdam
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 Erasmus University Medical Center Rotterdam filed Critical Erasmus University Medical Center Rotterdam
Publication of WO2011000516A2 publication Critical patent/WO2011000516A2/en
Publication of WO2011000516A3 publication Critical patent/WO2011000516A3/en

Links

Classifications

    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/5044—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics involving specific cell types
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/689—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to pregnancy or the gonads
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Chemical & Material Sciences (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Molecular Biology (AREA)
  • Cell Biology (AREA)
  • Analytical Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Pathology (AREA)
  • General Physics & Mathematics (AREA)
  • Food Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Pregnancy & Childbirth (AREA)
  • Reproductive Health (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

The invention relates to a method for identifying inhibitors of γ-globin gene expression suppression, to suppressors of γ-globin gene expression, and to the inhibitors as such.
PCT/EP2010/003854 2009-06-29 2010-06-29 Gamma globin therapy WO2011000516A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GB0911215A GB0911215D0 (en) 2009-06-29 2009-06-29 Gamma globin therapy
GB0911215.2 2009-06-29

Publications (2)

Publication Number Publication Date
WO2011000516A2 WO2011000516A2 (en) 2011-01-06
WO2011000516A3 true WO2011000516A3 (en) 2011-04-14

Family

ID=41008427

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2010/003854 WO2011000516A2 (en) 2009-06-29 2010-06-29 Gamma globin therapy

Country Status (2)

Country Link
GB (1) GB0911215D0 (en)
WO (1) WO2011000516A2 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018126499A1 (en) * 2017-01-05 2018-07-12 深圳迈瑞生物医疗电子股份有限公司 Method for preparing reticulocyte simulated particle and platelet simulated particle, and quality control material
CN111638261B (en) * 2020-04-17 2023-04-07 融智生物科技（青岛）有限公司 Computing equipment, storage medium and thalassemia screening device and system

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007140335A2 (en) * 2006-05-25 2007-12-06 The Regents Of The University Of Michigan Screening methods and transgenic animals for treatment of beta-globin related diseases and conditions

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US5175385A (en) 1987-09-03 1992-12-29 Ohio University/Edison Animal Biotechnolgy Center Virus-resistant transgenic mice
US5175384A (en) 1988-12-05 1992-12-29 Genpharm International Transgenic mice depleted in mature t-cells and methods for making transgenic mice
US5464764A (en) 1989-08-22 1995-11-07 University Of Utah Research Foundation Positive-negative selection methods and vectors
AU633698B2 (en) 1990-01-12 1993-02-04 Amgen Fremont Inc. Generation of xenogeneic antibodies
RO115498B1 (en) 1993-10-29 2000-03-30 Univ Boston Method for treating neoplasm
US6403647B1 (en) 1996-07-26 2002-06-11 Susan P. Perrine Pulsed administration of compositions for the treatment of blood disorders
GB2344886B (en) 1999-03-10 2000-11-01 Medical Res Council Selection of intracellular immunoglobulins
US8273866B2 (en) 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
WO2003014960A2 (en) 2001-08-03 2003-02-20 Medical Research Council Method of identifying a consensus sequence for intracellular antibodies
AU2003219277A1 (en) 2002-03-14 2003-09-29 Medical Research Council Intracellular antibodies
GB0226727D0 (en) 2002-11-15 2002-12-24 Medical Res Council Intrabodies
ITTO20020684A1 (en) 2002-07-31 2004-02-01 Associazione Veneta Per La Lotta Alla Talassemia NEW USE OF ANGELICINA AND ITS STRUCTURAL ANALOGUES.
GB0226729D0 (en) 2002-11-15 2002-12-24 Medical Res Council Intracellular antibodies
WO2005011696A1 (en) 2003-08-01 2005-02-10 Fibrogen, Inc. Inhibitors of 2-oxoglutarate dioxygenase as gamma globin inducers

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007140335A2 (en) * 2006-05-25 2007-12-06 The Regents Of The University Of Michigan Screening methods and transgenic animals for treatment of beta-globin related diseases and conditions

Non-Patent Citations (8)

* Cited by examiner, † Cited by third party
Title
AERBAJINAI WULIN ET AL: "SCF induces gamma-globin gene expression by regulating downstream transcription factor COUP-TFII", BLOOD, vol. 114, no. 1, 28 April 2009 (2009-04-28), pages 187 - 194, XP002603796, ISSN: 0006-4971 *
GROSVELD FRANK ET AL: "Isolation and characterization of hematopoietic transcription factor complexes by in vivo biotinylation tagging and mass spectrometry", ANNALS OF THE NEW YORK ACADEMY OF SCIENCES NEW YORK ACAD SCIENCES, 2 EAST 63RD ST, NEW YORK, NY 10021 USA SERIES : ANNALS OF THE NEW YORK ACADEMY OF SCIENCES (ISSN 0077-8923(PRINT)), 2005, & 8TH COOLEYS ANEMIA SYMPOSIUM; LAKE BUENA VISTA, FL, USA; MARCH 17 -19, 2005, pages 55 - 67, XP002619604 *
HALEY JOHN D ET AL: "Identification and characterization of mechanistically distinct inducers of gamma-globin transcription", BIOCHEMICAL PHARMACOLOGY, PERGAMON, OXFORD, GB LNKD- DOI:10.1016/S0006-2952(03)00542-2, vol. 66, no. 9, 1 November 2003 (2003-11-01), pages 1755 - 1768, XP002460405, ISSN: 0006-2952 *
HARJU-BAKER S ET AL: "Silencing of <A>[gamma]-globin gene expression during adult definitive erythropoiesis mediated by GATA-1-FOG-1-Mi2 complex binding at the 566 GATA site", MOLECULAR AND CELLULAR BIOLOGY 200805 US LNKD- DOI:10.1128/MCB.01858-07, vol. 28, no. 10, May 2008 (2008-05-01), pages 3101 - 3113, XP002603795, ISSN: 0270-7306 *
O'NEILL DAVID W ET AL: "An Ikaros-containing chromatin-remodeling complex in adult-type erythroid cells", MOLECULAR AND CELLULAR BIOLOGY, vol. 20, no. 20, October 2000 (2000-10-01), pages 7572 - 7582, XP002603794, ISSN: 0270-7306 *
PACE BETTY S ET AL: "Understanding mechanisms of gamma-globin gene regulation to develop strategies for pharmacological fetal hemoglobin induction.A", DEVELOPMENTAL DYNAMICS, WILEY-LISS, INC., NEW YORK, NY, US LNKD- DOI:10.1002/DVDY.20802, vol. 235, no. 7, 1 July 2006 (2006-07-01), pages 1727 - 1737, XP002459823, ISSN: 1058-8388 *
POURFARZAD ET AL: "Characterization of human gamma-globin silencing complex(es) in order to re-activate gamma-globin expression for the treatment of beta-thalassemia and sickle cell anemia", BLOOD CELLS, MOLECULES AND DISEASES, LAJOLLA, US LNKD- DOI:10.1016/J.BCMD.2006.10.121, vol. 38, no. 2, 2 February 2007 (2007-02-02), pages 173 - 174, XP005862073, ISSN: 1079-9796 *
SANKARAN VIJAY G ET AL: "Human fetal hemoglobin expression is regulated by the developmental stage-specific repressor BCL11A.", SCIENCE (NEW YORK, N.Y.) 19 DEC 2008 LNKD- PUBMED:19056937, vol. 322, no. 5909, 19 December 2008 (2008-12-19), pages 1839 - 1842, XP002603797, ISSN: 1095-9203 *

Also Published As

Publication number Publication date
GB0911215D0 (en) 2009-08-12
WO2011000516A2 (en) 2011-01-06

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