Calmodulin inhibitors for the treatment of ribosomal disorders and ribosomapathies
AssigneeChildren's Medical Center Corporation
InventorsLeonard I. Zon, Alison M. Taylor
Life of this patent
Estimating the term…
Abstract
The present invention relates generally to methods, compositions and kits for treatment of ribosomal disorders and ribosomopathy, e.g. Diamond Blackfan anemia (DBA). In some embodiments, the invention relates to methods for the use of calmodulin inhibitors and calcium channel blockers for treatment of ribosomal disorders and ribosomopathy, e.g. Diamond Blackfan anemia (DBA).
Claims11
A method of treating a subject with Diamond Blackfan Anemia (DBA), comprising administering an effective amount of a phenothiazine compound or CGS-9343 (zaldaride maleate) or a functional derivative or analog thereof to the subject to decrease p53 or p21 in at least one of CD34+ cells, erythroid cells or erythroid differentiated cells in the subject.
Dependencies are inferred from the claim text.