CELL THERAPY FOR NEUROMETABOLIC DISORDERS

Title:

CELL THERAPY FOR NEUROMETABOLIC DISORDERS

Document Type and Number:

WIPO Patent Application WO2005016250

Kind Code:

A3

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.

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Inventors:

SHIHABUDDIN LAMYA (US)
NUMAN SUZANNE (US)
STEWART GREGORY (US)

Application Number:

PCT/US2004/022311

Publication Date:

May 11, 2006

Filing Date:

August 06, 2004

Export Citation:

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Assignee:

GENZYME CORP (US)
SHIHABUDDIN LAMYA (US)
NUMAN SUZANNE (US)
STEWART GREGORY (US)

International Classes:

A01N43/04; A61K31/70; A61K35/30; C12N5/00; C12N5/02; C12N5/0797; A61K35/12; A61K

Domestic Patent References:

WO2001060377A2	2001-08-23
WO2003092594A2	2003-11-13

Foreign References:

US6541218B1

2003-04-01

Other References:

ETO ET AL: "Novel Treatment for Neurophatic-Lysosomal Storage Diseases-Cell Therapy.", CURR MOL MED., vol. 2, 2002, pages 83 - 89, XP002978662
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: "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

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