Treatment or replacement therapy using transgenic stem cells delivered to the gut
OwnerM. Michael Wolfe, Lisa I. Jepeal +1inventors
InventorsM. Michael Wolfe, Lisa I. Jepeal, Michael O. Boylan
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Abstract
The present invention is directed to methods for hormone delivery to patients suffering from a condition associated with a hormone deficiency. The method involves transducing stem cells, such as bone marrow derived stem cells, with a hormone gene under the control of a cell-type specific promoter such as the glucose-responsive GIP promoter, such that the hormone gene is expressed only after the stem cells differentiate into the cells which express the cell-type specific promoter, and administering the stem cells to the patient. A preferred embodiment of the present invention is the use of GIP-insulin gene expression in K cells of the gut to treat diabetes.
Claims60
A method for selectively expressing a desired hormone in a host for treating or replacing hormone in the host, said method comprising:
- (a) transducing a population of stem cells with a DNA sequence containing a gene encoding said desired hormone or a gene encoding a synthetic enzyme for said hormone, wherein said gene is operably-linked to a cell type specific promoter;
- (b) administering said transduced stem cells to a host under conditions wherein at least some of said stem cells differentiate into cells of the type said cell type specific promoter is specific for (referred to, as differentiated stem cells);
- and
- (c) allowing said differentiated stem cells to express said desired hormone or a synthetic enzyme for said hormone in the host to treat or replace hormone in the host.
A method for selectively expressing a desired active or pharmaceutical agent comprising:
- (a) transducing a population of stem cells with a DNA sequence containing a gene encoding said desired active or pharmaceutical agent, wherein said gene is operably-linked to a cell type specific promoter;
- (b) administering said transduced stem cells to a host under conditions wherein at least some of said stem cells differentiate into cells of the type said cell type specific promoter is specific for (referred to, as differentiated stem cells);
- and
- (c) allowing said differentiated stem cells to express said desired expressing a desired active or pharmaceutical agent.
Differentiated transduced stem cells delivered to the gut of a host for attaching to the gut and selectively expressing a desired active or pharmaceutical agent while engrafted in the intestine, said differentiated transduced stem cells comprising
- (a) a DNA sequence containing a gene encoding said desired active or pharmaceutical agent, wherein said gene is operably-linked to a cell type specific promoter, and
- (b) a cell type specific promoter which is specific for the differentiated transduced stem cells, wherein said differentiated transduced stem cells, while engrafted in the intestine, have the ability to express said desired active or pharmaceutical agent.
A population of transduced stem cells suitable for engrafting in the intestine of a host and differentiating therein once engrafted for selectively expressing a desired active or pharmaceutical agent comprising
- a population of stem cells transduced with a DNA sequence containing a gene encoding a desired active or pharmaceutical agent, wherein said gene is operably-linked to a cell type specific promoter, and wherein at least some of said population of stem cells, once engrafted in the intestine of a host, have the ability to (a) differentiate into cells of the type for which said cell type specific promoter is specific and (b) express the desired active or pharmaceutical agent.
Dependencies are inferred from the claim text.