WO2002044355A3 - Protozoan expression systems for lysosomal storage disease genes - Google Patents

Protozoan expression systems for lysosomal storage disease genes Download PDF

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Publication number
WO2002044355A3
WO2002044355A3 PCT/US2001/044935 US0144935W WO0244355A3 WO 2002044355 A3 WO2002044355 A3 WO 2002044355A3 US 0144935 W US0144935 W US 0144935W WO 0244355 A3 WO0244355 A3 WO 0244355A3
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WO
WIPO (PCT)
Prior art keywords
lysosomal storage
storage disease
methods
devices
expression systems
Prior art date
Application number
PCT/US2001/044935
Other languages
French (fr)
Other versions
WO2002044355A9 (en
WO2002044355A2 (en
Inventor
Dennis E Vaccaro
Stephen M Beverley
Jonathan H Lebowitz
Deborah Schmiel
John Maga
Original Assignee
Symbiontics Inc
Dennis E Vaccaro
Stephen M Beverley
Jonathan H Lebowitz
Deborah Schmiel
John Maga
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 Symbiontics Inc, Dennis E Vaccaro, Stephen M Beverley, Jonathan H Lebowitz, Deborah Schmiel, John Maga filed Critical Symbiontics Inc
Priority to AU2002241540A priority Critical patent/AU2002241540A1/en
Publication of WO2002044355A2 publication Critical patent/WO2002044355A2/en
Publication of WO2002044355A9 publication Critical patent/WO2002044355A9/en
Publication of WO2002044355A3 publication Critical patent/WO2002044355A3/en

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00—Preparation of peptides or proteins
    • C12P21/02—Preparation of peptides or proteins having a known sequence of two or more amino acids, e.g. glutathione
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01031—Beta-glucuronidase (3.2.1.31)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01052—Beta-N-acetylhexosaminidase (3.2.1.52)

Landscapes

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

Abstract

Methods and devices are provided for expressing and secreting gene products from protozoa. The methods and devices are useful for expressing and isolating lysosomal storage disease enzymes from protozoa grown in culture, and particularly from trypanosomatids. The post-translational modification of isolated expression products can be adapted for administration to mammalian organisms. In addition, expression products can be isolated from serum free cultures thereby avoiding contamination by infectious agents such as prions. The methods and devices are also useful for delivering expression products such as lysosomal storage disease enzymes to mammalian organisms in vivo.
PCT/US2001/044935 2000-11-30 2001-11-30 Protozoan expression systems for lysosomal storage disease genes WO2002044355A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2002241540A AU2002241540A1 (en) 2000-11-30 2001-11-30 Protozoan expression systems for lysosomal storage disease genes

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US25044600P 2000-11-30 2000-11-30
US25044400P 2000-11-30 2000-11-30
US60/250,446 2000-11-30
US60/250,444 2000-11-30
US29028101P 2001-05-11 2001-05-11
US60/290,281 2001-05-11

Publications (3)

Publication Number Publication Date
WO2002044355A2 WO2002044355A2 (en) 2002-06-06
WO2002044355A9 WO2002044355A9 (en) 2003-02-06
WO2002044355A3 true WO2002044355A3 (en) 2003-07-24

Family

ID=27400321

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2001/044935 WO2002044355A2 (en) 2000-11-30 2001-11-30 Protozoan expression systems for lysosomal storage disease genes

Country Status (2)

Country Link
AU (1) AU2002241540A1 (en)
WO (1) WO2002044355A2 (en)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
IL158623A0 (en) 2001-04-30 2004-05-12 Symbiontics Inc Subcellular targeting of therapeutic proteins
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
ATE446077T1 (en) * 2002-05-02 2009-11-15 Univ Rochester VECTORS CONTAINING BOTH ISOFORMS OF BETA-HEXOSAMINIDASE
US7261887B2 (en) 2002-11-12 2007-08-28 Rosalind Franklin University Of Medicine And Science Leishmania as carriers for the delivery of proteins and peptides
US20050244400A1 (en) 2004-02-10 2005-11-03 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
JP2009501128A (en) 2005-01-20 2009-01-15 ユニバーシティー オブ ロチェスター Compositions and methods for the study and treatment of inflammatory diseases and disorders
HUE034850T2 (en) 2008-05-07 2018-03-28 Biomarin Pharm Inc Lysosomal targeting peptides and uses thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1998011245A1 (en) * 1996-09-12 1998-03-19 Symbiontics, Inc. Sustained delivery device and methods of making and using the same
WO2000058483A2 (en) * 1999-03-26 2000-10-05 Washington University Protozoan expression system

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1998011245A1 (en) * 1996-09-12 1998-03-19 Symbiontics, Inc. Sustained delivery device and methods of making and using the same
WO2000058483A2 (en) * 1999-03-26 2000-10-05 Washington University Protozoan expression system

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
BURKE B ET AL: "Macrophages in gene therapy: Cellular delivery vehicles and in vivo targets.", JOURNAL OF LEUKOCYTE BIOLOGY, vol. 72, no. 3, September 2002 (2002-09-01), September, 2002, pages 417 - 428, XP009003789, ISSN: 0741-5400 *
MEIKLE PETER J ET AL: "Prevalence of lysosomal storage disorders.", JAMA (JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION), vol. 281, no. 3, 20 January 1999 (1999-01-20), pages 249 - 254, XP009003825, ISSN: 0098-7484 *
VACCARO D E: "Symbiosis therapy: the potential of using human protozoa for molecular therapy.", MOLECULAR THERAPY: THE JOURNAL OF THE AMERICAN SOCIETY OF GENE THERAPY. UNITED STATES DEC 2000, vol. 2, no. 6, December 2000 (2000-12-01), pages 535 - 538, XP002227144, ISSN: 1525-0016 *
VOTH BEVAN R ET AL: "Differentially expressed Leishmania major gp63 genes encode cell surface leishmanolysin with distinct signals for glycosylphosphatidylinositol attachment.", MOLECULAR AND BIOCHEMICAL PARASITOLOGY, vol. 93, no. 1, 15 May 1998 (1998-05-15), pages 31 - 41, XP002227143, ISSN: 0166-6851 *

Also Published As

Publication number Publication date
WO2002044355A9 (en) 2003-02-06
WO2002044355A2 (en) 2002-06-06
AU2002241540A1 (en) 2002-06-11

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