Abstract: Provided herein are N-(2-aminophenyl)-prop-2-enamide derivatives, such as those of Formula (I), methods for the synthesis thereof, and uses thereof in the treatment of cancer, such as SALL4-expressing cancer, in a cell or subject in need thereof.

Abstract: Provided herein are methods and agents for gene specific demethylation and/or activation. Oligonucleotide constructs are provided, the oligonucleotide constructs including: [1] a targeting portion having sequence complementarity and binding affinity with a region of genomic DNA within a gene, near a gene, or both; and [2] a single guide RNA (sgRNA) scaffold portion, wherein a tetra-loop portion of the sgRNA is modified and includes an R2 stem loop of DNMT1-interacting RNA (DiR), and wherein a stem loop 2 portion of the sgRNA is modified and includes an R5 step loop of DiR. The oligonucleotide constructs may be used, together with deactivated (dead) Cas9 (dCas9) for providing gene specific demethylation and/or activation of gene(s) of interest in a cell or subject in need thereof.

Abstract: The present invention relates to chimeric RNA oligonucleotides that are single-stranded oligonucleotides. These compounds are capable of targeting particular genes and reducing DNA methyltransferase activity. Accordingly, these compounds are particularly useful in the treatment of disease associated with aberrant DNA methyltransferase activity, such as cancer or a genetic disorder.

Abstract: The present invention relates to chimeric RNA oligonucleotides that are single-stranded oligonucleotides. These compounds are capable of targeting particular genes and reducing DNA methyltransferase activity. Accordingly, these compounds are particularly useful in the treatment of disease associated with aberrant DNA methyltransferase activity, such as cancer or a genetic disorder.

Abstract: The present invention relates to chimeric RNA oligonucleotides that are single-stranded oligonucleotides. These compounds are capable of targeting particular genes and reducing DNA methyltransferase activity. Accordingly, these compounds are particularly useful in the treatment of disease associated with aberrant DNA methyltransferase activity, such as cancer or a genetic disorder.

Abstract: The subject application relates to a hematopoietic stem cell specific promoter and to a hematopoietic stem cell specific enhancer and to uses therefor. The invention also includes a hematopoietic stem cell specific promoter-heterologous gene construct, where the expression of the heterologous gene is under the control of the myeloid cell type specific promoter. Additionally, the application is drawn to a method of expressing a heterologous gene in a hematopoietic stem cell. The invention further includes a method of expressing a selected heterologous gene product in hematopoietic stem cells in an individual. The invention also includes a transgenic non-human mammal in which the hematopoietic stem cells express a protein encoded by a heterologous gene. Finally the invention includes a method for identifying factors which can regulate hematopoietic stem cell specific transcription.

Abstract: The subject application is drawn to a method of transfecting a myeloid cell line and a method of producing a selected product in a myeloid cell. The invention also includes myeloid cell specific promoters and enhancers, and constructs which contain these promoters and enhancers. The invention further includes a myeloid cell specific promoter-heterologous gene construct, where the expression of the heterologous gene is under the control of the myeloid cell specific promoter. The invention also includes a transgenic non-human mammal in which myeloid cells express a protein encoded by a heterologous gene. Finally the invention includes a method for identifying factors that can regulate myeloid cell specific transcription.

Abstract: A recombinant DNA molecule consisting of segments of DNA from different genomes which have been joined end-to-end outside of living cells, and the progeny of such a recombinant DNA molecule, which recombinant DNA molecule (1) has the capacity to infect a host cell and to be maintained therein, and (2) comprises a DNA coding sequence selected from the group consisting of:(a) the CLCP-encoding sequence shown in FIG. 1,(b) a DNA sequence which hybridizes to the CLCP-encoding sequence shown in FIG. 1, and which encodes a polypeptide which (i) cross-reacts with an antibody to CLCP or to denatured CLCP, (ii) exhibits lysophospholipase activity, and (iii) forms dipyramidal crystals under conditions permitting CLC formation; and(c) a DNA sequence encoding a polypeptide encoded by any of the foregoing DNA sequences.