Abstract: Described are compositions and methods for targeting tumor associated transcription factors (e.g., PU.1) using IncRNA, constructs comprising IncRNA, and CRISPR/Cas systems, and polynucleotides encoding IncRNA, constructs comprising IncRNA, and CRISPR/Cas systems, vectors containing the polynucleotides, viral or non-viral delivery vehicles containing the vectors, and compositions (e.g., pharmaceutical compositions) containing the same for use in methods treatment.

Abstract: An aptamer, capable of inhibiting DNA methyltransferase 1 (DNMT1) for use in therapy of diseases characterised by aberrant DNA methylation, e.g. cancer. Method for identifying inhibitors of DNA methyltransferase. An aptamer, capable of inhibiting DNA methyltransferase 1 (DNMT1) for use in therapy of diseases characterised by aberrant DNA methylation, e.g. cancer. SELEX method for identifying aptamers of DNA methyltransferase optionally using 2-fluoro-pyrimindine nucleotide derivatives.

Abstract: Isolated peptides and pharmaceutical compositions comprising isolated peptides that bind to retinoblastoma binding protein 4 (RBBp4) and block the Spalt-Like Transcription Factor 4 (SALL4)-RBBp4 interaction are described. Methods of inhibiting binding of SALL4 to RBBp4 and methods of treating a subject having a disorder mediated by a dysregulation of SALL4 are also described.

Abstract: Described herein are methods for genetically modifying expanded hematopoietic cells comprising obtaining a quantity of hematopoietic cells, culturing the quantity of hematopoietic cells in the presence of at least one histone deacetylase inhibitor (HDACi) and at least one growth factor to expand the hematopoietic cells, and contacting the expanded hematopoietic cells with a nucleic acid sequence, wherein the nucleic acid sequence modifies the quantity of expanded hematopoietic cells. Further described herein are compositions comprising the genetically modified hematopoietic cells produced by these methods.

Abstract: The present invention provides, in certain embodiments, isolated peptides and pharmaceutical compositions comprising isolated peptides that bind to retinoblastoma binding protein 4 RBBp4 such that, the binding blocks the SALL4-RBBp4 interaction. Methods of inhibiting binding of SALL4 with RBBp4 and methods for treating a subject having a disorder mediated by a dysregulation of SALL4 are also provided.

Abstract: Described herein are methods and compositions which lead to the efficient ex vivo expansion of hematopoietic cells, such as hematopoietic stem cells (HSCs) and hematopoietic stem and progenitor cells HSPCs. Using combinations of small molecule drugs and cytokines/growth factors/grown factors targeting epigenetic status in cells, significant improvements in the expansion of cells was observed, including cells isolated from human cord blood or peripheral mobilized stem/progenitor cells. Multiple genes implicated in HSPC function were unperturbed, and efficiency of genomic editing using lentivirus was greatly enhanced following treatment. These novel approaches could be used therapeutically in a variety of hematopoietic transplantation settings, in addition to benefiting gene therapy techniques.

Abstract: Described are methods of treating a solid tumor which expresses Spalt-Like Transcription Factor 4 (SALL4) and Phosphatase and Tensin Homolog (PTEN) in an individual in need thereof, comprising administering to the individual an effective amount of a composition that inhibits SALL4. In addition, the invention is directed to methods of detecting an aggressive cancer in an individual in need thereof as well as methods of detecting a poor prognosis of a patient with cancer in an individual in need thereof.

Abstract: The invention is directed to methods of treating a solid tumor which expresses SALL4 and Phosphatase and Tensin Homolog (PTEN) in an individual in need thereof, comprising administering to the individual an effective amount of a composition that inhibits SALL4. In addition, the invention is directed methods of method of detecting an aggressive cancer in an individual in need thereof comprising detecting whether one or more cancer cells of the individual expresses SALL4, wherein if SALL4 is detected in the one or more cancer cells, then an aggressive cancer is detected in the individual, and methods of method of detecting a poor prognosis of a patient with cancer in an individual in need thereof, comprising detecting whether one or more cancer cells of the patient expresses SALL4, wherein if SALL4 is detected in the one or more cancer cells of the patient, then a poor prognosis is detected in the patient.