PAPD5 INHIBITION AS A TREATMENT FOR DYSKERATOSIS CONGENITA, APLASTIC ANEMIA AND MYELODYSPLASTIC SYNDROME CAUSED BY REDUCED TELOMERASE RNA LEVELS

摘要

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.