Therapeutic vulnerability of multiple myeloma to MIR17PTi, a first-in-class inhibitor of pri-miR-17-92

Author:

Morelli Eugenio12,Biamonte Lavinia1,Federico Cinzia13,Amodio Nicola1,Di Martino Maria Teresa1,Gallo Cantafio Maria Eugenia1,Manzoni Martina45ORCID,Scionti Francesca1,Samur Mehmet Kemal2,Gullà Annamaria12,Stamato Maria Angelica1,Pitari Maria Rita1,Caracciolo Daniele1,Sesti Settimio6,Frandsen Niels M.7,Rossi Marco1,Neri Antonino45,Fulciniti Mariateresa2,Munshi Nikhil C.28,Tagliaferri Pierosandro1,Tassone Pierfrancesco1

Affiliation:

1. Department of Experimental and Clinical Medicine, Magna Graecia University, Salvatore Venuta University Campus, Catanzaro, Italy;

2. Jerome Lipper Multiple Myeloma Center, Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA;

3. Cancer Biology Division, Department of Radiation Oncology, School of Medicine, Washington University in St. Louis, St. Louis, MO;

4. Department of Oncology and Oncoematology, University of Milan, Milan, Italy;

5. UOC Ematologia, Fondazione Cà Granda IRCCS, Ospedale Maggiore Policlinico, Milan, Italy;

6. Department of Biology, Ecology and Earth Science, University of Calabria, Cosenza, Italy;

7. Exiqon A/S, Vedbaek, Denmark; and

8. VA Boston Healthcare System, Boston, MA

Abstract

Key Points First-in-class MIR17PTi enables 1-shot downregulation of miR-17-92 in vitro and in vivo, with favorable pharmacokinetic profile. MIR17PTi affects homeostatic MYC/miR-17-92 FFLs in MM cells, resulting in strong anti-MM activity.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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