Abstract: Materials and methods for treating a patient to express a therapeutic agent comprising administering a Kupffer cell-suppressing substance in combination with a vehicle for introducing, into the patient, an exogenous nucleic acid comprising a sequence for expression of the agent.

Abstract: Human or humanized tissues and organs suitable for transplant are disclosed herein. Gene editing of a host animal provides a niche for complementation of the missing genetic information by donor stem cells. Editing of a host genome to knock out or disrupt genes responsible for the growth and/or differentiation of a target organ and injecting that animal at an embryo stage with donor stem cells to complement the missing genetic information for the growth and development of the organ. The result is a chimeric animal in which the complemented tissue (human/humanized organ) matches the genotype and phenotype of the donor. Such organs may be made in a single generation and the stem cell may be taken or generated from the patient's own body. As disclosed herein, it is possible to do so by simultaneously editing multiple genes in a cell or embryo creating a “niche” for the complemented tissue.

Abstract: Materials and methods for treating a patient to express a therapeutic agent comprising administering a Kupffer cell-suppressing substance in combination with a vehicle for introducing, into the patient, an exogenous nucleic acid comprising a sequence for expression of the agent.

Abstract: Human or humanized tissues and organs suitable for transplant are disclosed herein. Gene editing of a host animal provides a niche for complementation of the missing genetic information by donor stem cells. Editing of a host genome to knock out or disrupt genes responsible for the growth and/or differentiation of a target organ and injecting that animal at an embryo stage with donor stem cells to complement the missing genetic information for the growth and development of the organ. The result is a chimeric animal in which the complemented tissue (human/humanized organ) matches the genotype and phenotype of the donor. Such organs may be made in a single generation and the stem cell may be taken or generated from the patient's own body. As disclosed herein, it is possible to do so by simultaneously editing multiple genes in a cell or embryo creating a “niche” for the complemented tissue.

Abstract: Materials and methods for treating a patient to express a therapeutic agent comprising administering a Kupffer cell-suppressing substance in combination with a vehicle for introducing, into the patient, an exogenous nucleic acid comprising a sequence for expression of the agent.

Abstract: The present invention is directed to improved transposons and transposases. The present invention also includes gene transfer systems, methods of using the transposons and transposases, and compositions including the transposons and transposases.

Abstract: Some aspects of the application describe materials and methods for making a molecular tether. A molecular tether, in certain embodiments, includes a target-DNA-binding domain having a specific binding affinity for a target-DNA segment in a host chromosome, a carrier-binding domain that specifically binds to a DNA segment on a carrier, and a spacer covalently bonded to the target DNA-binding domain and the carrier-binding domain.

Abstract: The present invention provides for transposon vectors encoding expression control region-traps and gene-traps. Also provided are dicistronic vectors. Certain embodiments of the invention contain internal ribosome entry sites.

Abstract: This invention relates to a system for introducing nucleic acid into the DNA of a cell. The system includes the use of a member of the SB family of transposases (SB) or nucleic acid encoding the transposase and a nucleic acid fragment that includes a nucleic acid sequence with flanking inverted repeats. The transposase recognizes at least a portion of an inverted repeats and incorporates the nucleic acid sequence into the DNA. Methods for use of this system are discussed.

Abstract: Certain embodiments are directed to using an insulator element in a transposon having at least one transcriptional unit and at least one insulator element. The transcriptional unit(s) may be flanked by at least one insulator element on each side. The transcriptional unit may include an exogenous nucleic acid for introduction into a cell, e.g., DNA encoding a marker molecule. The insulator element may include a binding site for a CTCF protein. And, for example, a transcriptional unit may be disposed between a first insulator element and a second insulator element, and the first insulator element and the second insulator element may be disposed between inverted repeats of a transposon. The exogenous nucleic acid may be, e.g., DNA encoding an antisense RNA or siRNA.

Abstract: The present invention is directed to improved transposons and transposases. The present invention also includes gene transfer systems, methods of using the transposons and transposases, and compositions including the transposons and transposases.

Abstract: The application is related to the field of nucleic acids with identified utility, and more particularly, to genes, related nucleic acids, their complements, polypeptides, and methods of using the same for blood vessel, cartilage, and bone formation, as well as inhibition thereof. The application describes discoveries made using the zebrafish embryo technique, as well as other techniques that are described herein. The discoveries include genes, related nucleic acids, and their complements, as well as sequences, polypeptides, other molecules, and methods for using them, e.g., TDE1, PTV, MOESIN, and HKE4. Also described are polypeptide products, inhibition of expression, administration of materials and products, screening procedures, and techniques for making drugs. Moreover, uses of these discoveries in appropriate contexts are set forth.

Abstract: The present invention provides for transposon vectors encoding expression control region-traps and gene-traps. Also provided are dicistronic vectors. Certain embodiments of the invention contain internal ribosome entry sites.

Abstract: This invention relates to a system for introducing nucleic acid into the DNA of a cell. The system includes the use of a member of the SB family of transposases (SB) or nucleic acid encoding the transposase and a nucleic acid fragment that includes a nucleic acid sequence with flanking inverted repeats. The transposase recognizes at least a portion of an inverted repeats and incorporates the nucleic acid sequence into the DNA. Methods for use of this system are discussed.

Abstract: This invention relates to a system for introducing nucleic acid into the DNA of a cell. The system includes the use of a member of the SB family of transposases (SB) or nucleic acid encoding the transposase and a nucleic acid fragment that includes a nucleic acid sequence with flanking inverted repeats. The transposase recognizes at least a portion of an inverted repeats and incorporates the nucleic acid sequence into the DNA. Methods for use of this system are discussed.

Abstract: The present invention provides cationic polymers that include a primary amine and a targeting group covalently bound to the primary amine, wherein the targeting group targets a cell of interest by interacting with the surface of the cell. The invention also provides molecular complexes that include a polyethyleneimine and a targeting group covalently bound to a primary amine of the polyethyleneimine, and a biologically active compound. The invention further provides methods for delivering a biologically active compound to a vertebrate cell.

Abstract: This invention relates to a system for introducing nucleic acid into the DNA of a cell. The system includes the use of a member of the SB family of transposses (SB) or nucleic acid encoding the transposase and a nucleic acid fragment that includes a nucleic acid sequence with flanking inverted repeats. The transposase recognizes at least a portion of an inverted repeats and incorporates the nucleic acid sequence into the DNA. Methods for use of this system are discussed.