COMPOSITIONS AND METHODS FOR CANCER AND CANCER STEM CELL DETECTION AND ELIMINATION

摘要

In alternative embodiments, the invention provides compositions and methods for inhibiting or ablating cancer stem cells. In alternative embodiments, the invention provides compositions and methods for inhibiting the action of double-stranded RNA-specific adenosine deaminases, or ADAR, enzymes. In alternative embodiments, the invention provides compositions and methods for treating, ameliorating or preventing diseases and conditions responsive to the inhibition of cell differentiation and/or self-renewal of dysfunctional cells, cancer cells, leukemia cells, hematopoietic stem cells or cancer stem cells, e.g., leukemia or Chronic Myeloid Leukemia (CML). In alternative embodiments, the invention provides compositions and methods for inhibiting a Sonic Hedgehog (Shh) pathway, e.g., by using a Smoothened (SMO) protein inhibitor. In alternative embodiments, the invention provides compositions and methods for measuring or determining, or predicting, chronic myelogenous leukemia (CML) progression, Leukemic Stem Cell (LSC) generation and/or tyrosine kinase inhibitor resistance. In alternative embodiments, the invention provides compositions and methods for determining or measuring the effectiveness of a treatment, a drug, a therapy or a diet for eliminating, killing or reducing the amounts of a leukemic stem cell (LSC) or cells.

主权项

1. A method for treating, ameliorating or preventing diseases and conditions responsive to the inhibition or slowing of cell differentiation, or self-renewal or self-renewal capacity of dysfunctional cells, cancer cells, leukemia cells, hematopoietic stem cells or cancer stem cells, comprising,
(a) providing a composition that inhibits or slows the expression of or the activity of: an ADAR1 gene (adenosine deaminase acting on RNA 1), an ADAR1 gene product, an ADAR1 transcript, and/or an ADAR1 polypeptide; and (b) administering a sufficient amount of the composition to an individual in need thereof, wherein a sufficient amount comprises the inhibition or slowing of cell differentiation, or self-renewal or self-renewal capacity of dysfunctional cells, cancer cells, leukemia cells, hematopoietic stem cells or cancer stem cells.