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

Summary
Osteogenesis Imperfecta (OI), also known as brittle bone disease, is a prevalent rare skeletal condition. With limited treatment options available and no known cure, individuals with OI endure numerous bone fractures throughout their lives.
To address the urgent need for an OI cure, MORBIUS aims to create a gene therapy: employ a cutting-edge AAV-mediated gene editing, to curatively repair the genetic defect in a model of patient bone tissue (‘mini brittle bone’). Within MORBIUS I will: 1) construct ‘mini brittle bones’ - an OI patient-derived induced stem cell-based bone organoid system, and 2) repair the genetic defect in the ‘mini brittle bones’ with the AAV-based CRISPR/Cas and GeneRide strategy. To this end, with access to one of the largest OI cell line biobanks in the world, I will combine highly innovative induced stem cell development techniques with the existing expertise in fabrication of bone organoids of the hosting team and genome modification competence of secondment host.
Specifically, I will optimize a method of OI patient-derived induced mesenchymal stem cells embedding in a decellularized extracellular matrix to promote their natural differentiation and formation of ‘mini brittle bones’. Crafted ‘mini brittle bones’ will be transduced with bone-directed AAV vector carrying CRISPR/Cas and GeneRide systems to correct patient’s genetic defect by targeted insertion of a ‘donor’ sequence.
In synergy with gene therapy research, during a non-academic placement with an OI patient organization, an interactive multilingual educational webapp on OI gene therapy and genetics will be developed. This will ensure early knowledge sharing, awareness, building of trust, and encouragement of OI individuals to actively engage in gene therapy advancements.
MORBIUS outcomes will enhance the quality of life for individuals with OI and other rare bone disorders, but introduction of effective gene editing, bone organoid modeling, and knowledge sharing methods.
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More information & hyperlinks
Web resources: https://cordis.europa.eu/project/id/101151143
Start date: 01-01-2025
End date: 30-06-2027
Total budget - Public funding: - 226 441,00 Euro
Cordis data

Original description

Osteogenesis Imperfecta (OI), also known as brittle bone disease, is a prevalent rare skeletal condition. With limited treatment options available and no known cure, individuals with OI endure numerous bone fractures throughout their lives.
To address the urgent need for an OI cure, MORBIUS aims to create a gene therapy: employ a cutting-edge AAV-mediated gene editing, to curatively repair the genetic defect in a model of patient bone tissue (‘mini brittle bone’). Within MORBIUS I will: 1) construct ‘mini brittle bones’ - an OI patient-derived induced stem cell-based bone organoid system, and 2) repair the genetic defect in the ‘mini brittle bones’ with the AAV-based CRISPR/Cas and GeneRide strategy. To this end, with access to one of the largest OI cell line biobanks in the world, I will combine highly innovative induced stem cell development techniques with the existing expertise in fabrication of bone organoids of the hosting team and genome modification competence of secondment host.
Specifically, I will optimize a method of OI patient-derived induced mesenchymal stem cells embedding in a decellularized extracellular matrix to promote their natural differentiation and formation of ‘mini brittle bones’. Crafted ‘mini brittle bones’ will be transduced with bone-directed AAV vector carrying CRISPR/Cas and GeneRide systems to correct patient’s genetic defect by targeted insertion of a ‘donor’ sequence.
In synergy with gene therapy research, during a non-academic placement with an OI patient organization, an interactive multilingual educational webapp on OI gene therapy and genetics will be developed. This will ensure early knowledge sharing, awareness, building of trust, and encouragement of OI individuals to actively engage in gene therapy advancements.
MORBIUS outcomes will enhance the quality of life for individuals with OI and other rare bone disorders, but introduction of effective gene editing, bone organoid modeling, and knowledge sharing methods.

Status

SIGNED

Call topic

HORIZON-MSCA-2023-PF-01-01

Update Date

21-11-2024
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Horizon Europe
HORIZON.1 Excellent Science
HORIZON.1.2 Marie Skłodowska-Curie Actions (MSCA)
HORIZON.1.2.0 Cross-cutting call topics
HORIZON-MSCA-2023-PF-01
HORIZON-MSCA-2023-PF-01-01 MSCA Postdoctoral Fellowships 2023