Abstract: This disclosure provides a system for generating animal tissue with carbohydrate antigens that are compatible for transplantation into human patients. The tissue is inactivated homozygously for expression of ?(1,3)galactosyltransferase, and comprises a transgene for ?(1,2)fucosyltransferase. As a result, cell-surface N-acetyl lactosamine is not converted to the Gal?(1,3)Gal xenoantigen. Instead, it is converted to Fuc?(1,2)Gal, which is H substance, a self-antigen in humans. The tissue may also contain A or B-transferase, which will cause H substance to be converted into other ABO blood group antigens for compatibility with patients of the same blood type. This invention improves transplant compatibility of the xenograft tissue by lessening the risk of reactions resulting from xenoantigen and unconverted N-acetyl lactosamine acceptor determinants.

Abstract: This invention provides a system for producing differentiated cells from a stem cell population for use wherever a relatively homogenous cell population is desirable. The cells contain an effector gene under control of a transcriptional control element (such as the TERT promoter) that causes the gene to be expressed in relatively undifferentiated cells in the population. Expression of the effector gene results in expression of a cell-surface antigen that can be used to deplete the undifferentiated cells. Model effector sequences encode glycosyl transferases that synthesize carbohydrate xenoantigen or alloantigen, which can be used for immunoseparation or as a target for complement-mediated lysis. The differentiated cell populations produced are suitable for use in tissue regeneration and non-therapeutic applications such as drug screening.

Abstract: This invention provides a system for producing differentiated cells from a stem cell population for use wherever a relatively homogenous cell population is desirable. The cells contain an effector gene under control of a transcriptional control element (such as the TERT promoter) that causes the gene to be expressed in relatively undifferentiated cells in the population. Expression of the effector gene results in expression of a cell-surface antigen that can be used to deplete the undifferentiated cells. Model effector sequences encode glycosyl transferases that synthesize carbohydrate xenoantigen or alloantigen, which can be used for immunoseparation or as a target for complement-mediated lysis. The differentiated cell populations produced are suitable for use in tissue regeneration and non-therapeutic applications such as drug screening.

Abstract: This disclosure provides a system for specifically killing cancer cells which can be used in the course of human therapy. Vectors of the invention comprise an encoding sequence for a glycosyltransferase, under control of a tumor or tissue specific transcriptional control element, such as the promoter for telomerase reverse transcriptase. Exemplary glycosyltransferases are the A or B transferase enzymes, which cause the cancer cells to express ABO histo blood group allotypes against which humans have naturally occurring antibody. This provides for ongoing surveillance for newly emerging cells with a malignant phenotype.

Abstract: Described in this disclosure is a new process whereby cells of one tissue type can be reprogrammed to produce cells of a different tissue type. Cells from a human donor are reprogrammed by culturing adjacent to primate pluripotent stem cells (in an undifferentiated or newly differentiated state) or in an environment supplemented by components taken from pPS cells. Simultaneously or in a subsequent step, the donor cells can be treated in a manner that enhances differentiation towards a different tissue type. In this manner, patients in need of tissue regeneration can be treated with cells differentiated and reprogrammed from their own autologous cell donation.

Abstract: It has been discovered that the specificity of multiple transcriptional regulatory elements can be combined to make vector systems that selectively target cancer cells. The promoter for telomerase reverse transcriptase (TERT) can be combined in a remarkably synergistic fashion with another promoter that has expression restricted to cancer cells or a particular tissue type. The two promoters work synergistically for exquisite targeting of the malignant cells—where it causes cell lysis while leaving neighboring healthy cells intact.

Abstract: This disclosure provides a system for generating animal tissue with carbohydrate antigens that are compatible for transplantation into human patients. The tissue is inactivated homozygously for expression of &agr;(1,3)galactosyltransferase, and comprises a transgene for &agr;(1,2)fucosyltransferase. As a result, cell-surface N-acetyl lactosamine is not converted to the Gal&agr;(1,3)Gal xenoantigen. Instead, it is converted to Fuc&agr;(1,2)Gal, which is H substance, a self-antigen in humans. The tissue may also contain A or B-transferase, which will cause H substance to be converted into other ABO blood group antigens for compatibility with patients of the same blood type. This invention improves transplant compatibility of the xenograft tissue by lessening the risk of reactions resulting from xenoantigen and unconverted N-acetyl lactosamine acceptor determinants.

Abstract: This invention provides a system for producing differentiated cells from a stem cell population for use wherever a relatively homogenous cell population is desirable. The cells contain an effector gene under control of a transcriptional control element (such as the TERT promoter) that causes the gene to be expressed in relatively undifferentiated cells in the population. Expression of the effector gene results in expression of a cell-surface antigen that can be used to deplete the undifferentiated cells. Model effector sequences encode glycosyl transferases that synthesize carbohydrate xenoantigen or alloantigen, which can be used for immunoseparation or as a target for complement-mediated lysis. The differentiated cell populations produced are suitable for use in tissue regeneration and non-therapeutic applications such as drug screening.

Abstract: This disclosure provides a system for specifically killing cancer cells which can be used in the course of human therapy. Vectors of the invention comprise an encoding sequence for a glycosyltransferase, under control of a tumor or tissue specific transcriptional control element, such as the promoter for telomerase reverse transcriptase. Exemplary glycosyltransferases are the A or B transferase enzymes, which cause the cancer cells to express ABO histo blood group allotypes against which humans have naturally occurring antibody. This provides for ongoing surveillance for newly emerging cells with a malignant phenotype.