WO2002044355A3 - Protozoan expression systems for lysosomal storage disease genes - Google Patents
Protozoan expression systems for lysosomal storage disease genes Download PDFInfo
- 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
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- lysosomal storage
- storage disease
- methods
- devices
- expression systems
- Prior art date
Links
Classifications
-
- 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
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)
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)
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 |
-
2001
- 2001-11-30 WO PCT/US2001/044935 patent/WO2002044355A2/en not_active Application Discontinuation
- 2001-11-30 AU AU2002241540A patent/AU2002241540A1/en not_active Abandoned
Patent Citations (2)
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)
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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