The present disclosure is directed to methods of inducing rejuvenation in a population of adult glial progenitor cells, and methods of treating a subject having a myelin deficiency. The method of inducing rejuvenation in a population of adult glial progenitor cells, may comprise: administering, to the population of adult glial progenitor cells, one or more nucleic acid molecules encoding microRNAs, wherein administering suppresses the signal transducer and activator of transcription 3 (STAT3) signaling pathway; and/or administering microRNAs, wherein administering suppresses the E2F transcription factor 6 (E2F6) signaling pathway; and/or administering microRNAs, wherein administering suppresses the Myc-associated factor X (MAX) signaling pathway, wherein said one or more nucleic acid molecules are administered in an amount sufficient to induce rejuvenation in the population of adult glial progenitor cells.

The present disclosure relates to AAV gene therapy vectors, AAV replicons, and pharmaceutical compositions for delivering a human HDAC8 gene to a subject for treating Cornelia de Lange Syndrome. In addition, methods of treatment and gene transfer are provided.

The present disclosure provides compositions with a modulating gene expression and methods for modulating transcription.

The present disclosure provides for methods and compositions for treating cancer. A subject having lymphoma is administered an EZH2 inhibitor and an HDAC inhibitor. The combination of the EZH2 inhibitor and the HDAC inhibitor produces a synergistic effect on the cancer compared to the effect of the EZH2 inhibitor or the HDAC inhibitor alone.

The present disclosure provides compositions with a modulating gene expression and methods for modulating transcription.

Disclosed herein are conditional siRNAs activatable by CBFβ-MYH11 oncogenic gene and use thereof for treating conditions such as acute myeloid leukemia (AML). The conditional siRNAs target MCL-1 or HDAC8.

The present disclosure is directed to methods of inducing rejuvenation in a population of adult glial progenitor cells, and methods of treating a subject having a myelin deficiency. The method of treating a subject having a myelin deficiency, may comprise expressing, in glial progenitor cells of the subject, one or more transcription factors selected from the group consisting of B-cell lymphoma/leukemia 11A (BCL11A), histone deacetylase 2 (HDAC2), histone-lysine N-methyltransferase EZH2 (EZH2), myc proto-oncogene protein (MYC), high mobility group protein HMGI-C (HMGA2), nuclear factor 1 B-type (NFIB), and transcriptional enhancer factor TEF-4 10 (TEAD2), wherein said expressing is effective to induce myelination in the subject, thereby treating the myelin deficiency.

A method for predicting a survival rate of a breast cancer patient, including: providing a biological sample of the breast cancer patient; measuring miR-125a-5p expression level in the biological sample; and comparing the miR-125a-5p expression level in the biological sample of the breast cancer patient with miR-125a-5p expression level in another biological sample of a reference breast cancer patient. When the miR-125a-5p expression level in the biological sample of the breast cancer patient is lower than that of the reference breast cancer patient, the survival rate of the breast cancer patient is lower than that of the reference breast cancer patient. When the miR-125a-5p expression level in the biological sample of the breast cancer patient is greater than that of the reference breast cancer patient, the survival rate of the breast cancer patient is greater than that of the reference breast cancer patient.

The present disclosure provides compositions with a modulating gene expression and methods for modulating transcription.

The present disclosure provides for methods and compositions for treating cancer. A subject having lymphoma is administered an EZH2 inhibitor and an HDAC inhibitor. The combination of the EZH2 inhibitor and the HDAC inhibitor produces a synergistic effect on the cancer compared to the effect of the EZH2 inhibitor or the HDAC inhibitor alone.