Compositions and methods for the treatment of lysosomal storage disorders
First Claim
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1. A method for providing a lysosomal enzyme to a subject comprising administering stem cells to the subject, wherein the stem cells can give rise to cells of endodermal, ectodermal and mesodermal lineages, and wherein the stem cells provide a lysosomal enzyme.
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Abstract
The present invention relates to methods for providing lysosomal enzymes to a subject by administering stem cells, preferably Multipotent Adult Progenitor Cells (MAPCs). The invention further relates to methods for treating lysosomal storage disorders by administering stem cells.
44 Citations
31 Claims
- 1. A method for providing a lysosomal enzyme to a subject comprising administering stem cells to the subject, wherein the stem cells can give rise to cells of endodermal, ectodermal and mesodermal lineages, and wherein the stem cells provide a lysosomal enzyme.
- 2. A method for treating a lysosomal storage disorder comprising administering stem cells to a subject having a lysosomal storage disorder, wherein the stem cells can give rise to cells of endodermal, ectodermal and mesodermal lineages, and wherein the stem cells, following administration, express an amount of lysosomal enzyme effective to treat said disorder.
- 3. A method for modulating accumulation of a substrate in a subject comprising providing an amount of a lysosomal enzyme to the subject effective to modulate said accumulation, wherein the lysosomal enzyme is provided by stem cells administered to said subject and wherein the stem cells can give rise to cells of endodermal, ectodermal and mesodermal lineages.
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Specification