Multivalent morpholino-based antisense system for CML (MOR4CML)

Project leader: Alena Gábelová
Project duration: 2016 - 2020

The proposed MOR4CML project is dedicated to an innovative, morpholino-based antisense concept for BCR-ABL silencing in patients with chronic myelogenous leukemia (CML). Due to its original design and mechanism of action, the suggested concept allows to significantly enhance both selectivity towards CML cells and sequence-mediated specificity towards BCR-ABL mRNA. It is expected that the presented interdisciplinary effort will overcome the main issues that have so far hindered introduction of BCR-ABL antisense-therapeutics into clinical practice. If successful, the proposed concept is also expected to provide a tremendous curative potential not only in CML but also in all diseases with known molecular basis, where a particular protein plays a causal role in disease pathophysiology.