MAPC administration for the treatment of lysosomal storage disorders
First Claim
1. A method for providing a lysosomal enzyme to a subject comprising administering stem cells to the subject, wherein the stem cells comprise isolated expanded human multipotent non-embryonic non-germ cells that can differentiate into at least one cell type of each of the endodermal, ectodermal, and mesodermal embryonic lineages and express telomerase, said cells having undergone at least 10-40 cell doublings in culture, 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.
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Citations
18 Claims
- 1. A method for providing a lysosomal enzyme to a subject comprising administering stem cells to the subject, wherein the stem cells comprise isolated expanded human multipotent non-embryonic non-germ cells that can differentiate into at least one cell type of each of the endodermal, ectodermal, and mesodermal embryonic lineages and express telomerase, said cells having undergone at least 10-40 cell doublings in culture, 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 comprise isolated expanded human multipotent non-embryonic non-germ cells that can differentiate into at least one cell type of each of the endodermal, ectodermal, and mesodermal embryonic lineages and express telomerase, said cells having undergone at least 10-40 cell doublings in culture, 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 comprise isolated expanded human multipotent non-embryonic non-germ cells that can differentiate into at least one cell type of each of the endodermal, ectodermal, and mesodermal embryonic lineages and express telomerase, said cells having undergone at least 10-40 cell doublings in culture.
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18. A method for modulating accumulation of a substrate in a subject comprising providing α
- -L-iduronidase to the subject in an amount effective to modulate said accumulation, wherein the α
-L-iduronidase is provided by stem cells administered to the subject and wherein the stem cells comprise isolated expanded human multipotent non-embryonic non-germ cells that can differentiate into at least one cell type of each of the endodermal, ectodermal, and mesodermal embryonic lineages and express telomerase and oct3/4, said cells having undergone at least 10-40 cell doublings in culture, wherein said enzyme is provided by systemic injection, and wherein said subject has Hurler'"'"'s syndrome.
- -L-iduronidase to the subject in an amount effective to modulate said accumulation, wherein the α
Specification