Abstract: Increased PARN as an indicator of a cancer involving loss or reduction in p53 function. PARN is also provided as a therapeutic target for treating a cancer involving loss or reduction in p53 function. Methods of treating a subject having a cancer involving loss or reduction in p53 function based on the level of PARN in a test sample obtained from the subject and administering an effective amount of a PARN inhibitor, alone or in conjunction with another chemotherapeutic agent, to the subject to treat the cancer.

Abstract: The invention includes methods and compositions for the treatment and prevention of a disease or condition resulting from dysregulation of USB1 and resulting expression of downstream miRNAs. In one preferred embodiment, the invention may include inhibiting the expression or activity of USB1 resulting in the downregulation of downstream miRNA expression. In alternative embodiments, the invention includes methods and compositions for the treatment and prevention of a disease or condition resulting from dysregulation of miRNAs downstream of USB1, namely through the inhibition of PAPD5 and/or PAPD7, resulting in the upregulation of said downstream miRNA expression.

Abstract: The present invention includes the novel therapeutic strategies, systems., and compositions for the treatment of telomere-associated disease or disorder through the inhibition of PAPD5/7. the present invention includes the novel therapeutic application of PAPD5 inhibitors for the treatment of disease conditions that implicate reduced telomerase RNA levels. In particular, the present invention includes the novel therapeutic application of PAPD5 inhibitors for the treatment of dyskeratosis congenita, aplastic anemia, and myelodysplastic syndrome. In one preferred embodiment, the present invention includes the novel therapeutic application of RG7834 for the treatment of disease conditions that implicate reduced telomerase RNA levels. In particular, the present invention includes the novel therapeutic application of RG7834 for the treatment of dyskeratosis congenita, aplastic anemia, and myelodysplastic syndrome.

Abstract: The invention includes methods and compositions for the treatment and prevention of a disease or condition resulting from dysregulation of USB1 and resulting expression of downstream miRNAs. In one preferred embodiment, the invention may include inhibiting the expression or activity of USB1 resulting in the downregulation of downstream miRNA expression. In alternative embodiments, the invention includes methods and compositions for the treatment and prevention of a disease or condition resulting from dysregulation of miRNAs downstream of USB1, namely through the inhibition of PAPD5 and/or PAPD7, resulting in the upregulation of said downstream miRNA expression.

Abstract: Increased PARN as an indicator of a cancer involving loss or reduction in p53 function. PARN is also provided as a therapeutic target for treating a cancer involving loss or reduction in p53 function. Methods of treating a subject having a cancer involving loss or reduction in p53 function based on the level of PARN in a test sample obtained from the subject and administering an effective amount of a PARN inhibitor, alone or in conjunction with another chemotherapeutic agent, to the subject to treat the cancer.