WO2005016250A3 - Cell therapy for neurometabolic disorders - Google Patents

Cell therapy for neurometabolic disorders Download PDF

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Publication number
WO2005016250A3
WO2005016250A3 PCT/US2004/022311 US2004022311W WO2005016250A3 WO 2005016250 A3 WO2005016250 A3 WO 2005016250A3 US 2004022311 W US2004022311 W US 2004022311W WO 2005016250 A3 WO2005016250 A3 WO 2005016250A3
Authority
WO
WIPO (PCT)
Prior art keywords
cell therapy
neurometabolic disorders
stem cells
neural stem
neurometabolic
Prior art date
Application number
PCT/US2004/022311
Other languages
French (fr)
Other versions
WO2005016250A2 (en
Inventor
Lamya Shihabuddin
Suzanne Numan
Gregory Stewart
Original Assignee
Genzyme Corp
Lamya Shihabuddin
Suzanne Numan
Gregory Stewart
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 Genzyme Corp, Lamya Shihabuddin, Suzanne Numan, Gregory Stewart filed Critical Genzyme Corp
Publication of WO2005016250A2 publication Critical patent/WO2005016250A2/en
Publication of WO2005016250A3 publication Critical patent/WO2005016250A3/en

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Classifications

    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0623—Stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/30—Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/10—Growth factors
    • C12N2501/11—Epidermal growth factor [EGF]
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/10—Growth factors
    • C12N2501/115—Basic fibroblast growth factor (bFGF, FGF-2)
    • 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
    • C12N2510/00—Genetically modified cells

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Neurosurgery (AREA)
  • Genetics & Genomics (AREA)
  • Neurology (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The disclosure pertains to methods for treating neurometabolic disorders including lysosomal storage diseases that affect the central nervous system such as, e.g., Niemann-Pick A disease. The disclosed methods involve administering nonimmortalized neural stem cells into the brain of a mammal, wherein the neural stem cells secrete a lysosomal hydrolase.
PCT/US2004/022311 2003-08-08 2004-08-06 Cell therapy for neurometabolic disorders WO2005016250A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US49365203P 2003-08-08 2003-08-08
US60/493,652 2003-08-08

Publications (2)

Publication Number Publication Date
WO2005016250A2 WO2005016250A2 (en) 2005-02-24
WO2005016250A3 true WO2005016250A3 (en) 2006-05-11

Family

ID=34193189

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2004/022311 WO2005016250A2 (en) 2003-08-08 2004-08-06 Cell therapy for neurometabolic disorders

Country Status (1)

Country Link
WO (1) WO2005016250A2 (en)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7927587B2 (en) 1999-08-05 2011-04-19 Regents Of The University Of Minnesota MAPC administration for the treatment of lysosomal storage disorders
AU2004296848A1 (en) 2003-12-04 2005-06-23 Regents Of The University Of Minnesota Compositions and methods for the treatment of lysosomal storage disorders
AU2007321928A1 (en) 2006-11-24 2008-05-29 Regents Of The University Of Minnesota Endodermal progenitor cells

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001060377A2 (en) * 2000-02-17 2001-08-23 Genzyme Corporation Methods for treatment of lysosomal storage diseases using biphosphonates
US6541218B1 (en) * 1991-05-03 2003-04-01 The Mount Sinai School Of Medicine Of The City University Of New York Acid sphingomyelinase protein and methods of treating type B Niemann-Pick disease
WO2003092594A2 (en) * 2002-04-30 2003-11-13 Duke University Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6541218B1 (en) * 1991-05-03 2003-04-01 The Mount Sinai School Of Medicine Of The City University Of New York Acid sphingomyelinase protein and methods of treating type B Niemann-Pick disease
WO2001060377A2 (en) * 2000-02-17 2001-08-23 Genzyme Corporation Methods for treatment of lysosomal storage diseases using biphosphonates
WO2003092594A2 (en) * 2002-04-30 2003-11-13 Duke University Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
ERLICH ET AL: "Fluorescence-Based Selection of Gene-Corrected Hematopoietic Stem and Progenitor Cells From Acid Sphingomyelinase-Deficient Mice: Implications for Niemann-Pick Disease Gene Therapy and the Development of Improved Stem Cell Gene Transfer Procedures.", BLOOD., vol. 93, 1999, pages 80 - 86, XP002997243 *
ETO ET AL: "Novel Treatment for Neurophatic-Lysosomal Storage Diseases-Cell Therapy.", CURR MOL MED., vol. 2, 2002, pages 83 - 89, XP002978662 *
ETO ET AL: "Treatment of Lysosomal storage disorders: Cell Therapy and gene therapy.", J INHERT METAB DIS., vol. 27, 2004, pages 411 - 415, XP002997244 *
MENG ET AL: "Brain Transplantation of Genetically Engineered Human Neural Stem Cells Globally Corrects Brain Lesions in the Mucopolysaccharidosis Type VII Mouse.", J NEUROSCI RES., vol. 74, 2003, pages 266 - 277, XP002997245 *

Also Published As

Publication number Publication date
WO2005016250A2 (en) 2005-02-24

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