MORBIUS - Cure for Miniature Brittle Bones: Treatment of Osteogenesis ImpeRfecta patient-derived Bone organoids with Innovative AAV-mediated Ultimate Gene-editing System

ABSTRACT

In the MORBIUS project, cutting-edge interdisciplinary expertise converges to revolutionize Osteogenesis Imperfecta (OI) research. Our ambitious MSCA-IF project aims to develop innovative bone organoids—mini brittle bones—using patient-derived induced stem cells. These 3D co-cultures replicate the natural processes of bone modeling and the unique pathophysiology of OI.

As a groundbreaking proof-of-concept, these engineered mini brittle bones serve as platforms for gene therapy approaches. By employing bone-targeting AAV vectors, we aim to either overexpress beneficial genes or edit disease-causing pathogenic variants, tailored to each patient’s genetic profile.

This initiative is a collaborative effort with leading-edge partners: IBIMA Plataforma Bionand in Spain for tissue engineering, Amsterdam UMC in The Netherlands for genetic and cell OI expertise, and UMASS in the US for bone-targeting gene therapies. We are also proud to partner with the Osteogenesis Imperfecta Federation Europe (OIFE), uniting OI organizations across Europe.

Furthermore, we are developing an educational web app to disseminate knowledge on OI gene therapy and genetics, empowering patients and fostering their active participation in therapeutic advancements.

The MORBIUS project is being conducted by Dr. Lidiia Zhytnik (IBIMA Plataforma Bionand), under the expert supervision of Prof. Ivan Duran (IBIMA Plataforma Bionand and The University of Malaga).

Follow us on this exciting journey to transform OI treatment through innovation and collaboration.

Participant Organisations

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MORBIUS

Project Information

Fellow

Dr. Liidia Zhytnik

Supervisors

Dr. Iván Durán

Dr. Fabiana Csukasi