Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases

a technology of lysosomal storage and enzyme replacement, applied in the field of therapeutics for lysosomal storage diseases, can solve the problems of frequency of administration, and achieve the effect of optimizing clinical benefit and minimizing disadvantages

Inactive Publication Date: 2002-07-18
MEEKER DAVID +1
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  • Abstract
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  • Claims
  • Application Information

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Benefits of technology

0030] This invention provides various combinations of enzyme replacement therapy, gene therapy, and small molecule therapy for the treatment of lysosomal storage diseases. According to the invention, several general approaches are provided. Each gen...

Problems solved by technology

A major disadvantage of ERT is the frequency of the administration requi...

Method used

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  • Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
  • Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
  • Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases

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Embodiment Construction

[0075] The therapeutic methods of the invention described herein provide treatment options for the practitioner faced with management of various lysosomal storage diseases, as described in detail below. More specifically, the invention relates to various combinations of enzyme replacement therapy and gene therapy for the treatment of lysosomal storage diseases.

[0076] A partial list of known lysosomal storage diseases that can be treated in accordance with the invention is set forth in Table 1, including common disease name, material stored, and corresponding enzyme deficiency (adapted from Table 38-4 of Kolodny et al., 1998, Id.).

1TABLE 1 Lysosomal Storage Diseases Disease Material Stored Enzyme Deficiency Sphingolipidoses Gaucher Glucocerebroside Glucocerebrosidase Niemann-Pick Sphingomyelin Sphingomyelinase Farber Ceramide Ceramidase G.sub.M1-gangliosidosis G.sub.M1-ganglioside, G.sub.M1-ganglioside-.beta.-- glycoprotein galactosidase G.sub.M2-gangliosidosis G.sub.M2-ganglioside, ...

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Abstract

This invention provides various combinations of enzyme replacement therapy, gene therapy, and small molecule therapy for the treatment of lysosomal storage diseases.

Description

[0001] This application claims priority of U.S. Provisional Application No. 60 / 212,377 filed Jun. 19, 2000, which is incorporated-by-reference herein in its entirety.1. FIELD OF THE INVENTION[0002] The present invention relates generally to the field of therapeutics for lysosomal storage diseases. More specifically, the invention relates to various combinations of enzyme replacement therapy, gene therapy, and small molecule therapy for the treatment of lysosomal storage diseases.2. BACKGROUND OF THE INVENTION[0003] Each of the over thirty known lysosomal storage diseases (LSDs) is characterized by a similar pathogenesis, namely, a compromised lysosomal hydrolase. Generally, the activity of a single lysosomal hydrolytic enzyme is reduced or lacking altogether, usually due to inheritance of an autosomal recessive mutation. As a consequence, the substrate of the compromised enzyme accumulates undigested in lysosomes, producing severe disruption of cellular architecture and various dise...

Claims

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Application Information

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IPC IPC(8): A61K31/445A61K38/47A61P3/00A61P9/10A61P43/00
CPCA61K31/445A61K38/47A61K2300/00A61P3/00A61P9/10A61P43/00
Inventor MEEKER, DAVIDCHENG, SENG H.
Owner MEEKER DAVID
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