Non-invasive growth factor gene therapy to promote remyelination in multiple sclerosis
Novel AAV-based gene therapy delivering CNS-wide growth factors to promote remyelination and functional recovery in multiple sclerosis, leveraging targets identified from MS patient brain tissue with spontaneous remyelination capacity.
Overview
This research proposes a non-invasive, CNS-wide gene therapy approach to promote remyelination in multiple sclerosis (MS). The therapy uses AAV-based gene delivery to express three growth factors identified through RNA sequencing of brain tissue from MS patients who exhibited high levels of spontaneous remyelination. By leveraging the body's natural repair mechanisms, this approach aims to restore myelin and recover function lost to progressive demyelination, addressing a critical unmet need beyond current anti-inflammatory treatments.
Technical specifications
- Novel target identification: Growth factors were discovered through RNAseq screening of lesions from MS patients showing significant spontaneous remyelination, ensuring biological relevance to human disease
- Non-invasive AAV gene delivery: Adeno-associated virus vectors enable CNS-wide expression of therapeutic genes without surgical intervention
- EAE animal model validation: The therapy is tested in experimental allergic encephalomyelitis, a well-established MS animal model, using functional and histological assessments
- Combinatorial approach: The research plan includes testing individual growth factors and combinations to identify optimal therapeutic regimens
- Growth factor identities: Specific growth factors can be disclosed under a non-disclosure agreement
Technology readiness level
The project is in preclinical development. Target identification and AAV delivery methodology have been established through prior work. The remaining validation includes generating AAV vectors for three growth factors, conducting longitudinal functional and histological studies in the EAE model, and evaluating combinatorial gene therapy approaches. The research is led by Prof. Joost Verhaagen and Prof. Inge Huitinga at the Netherlands Institute for Neuroscience.
About Netherlands Institute for Neuroscience
NIN is a focused research institute of the Royal Netherlands Academy of Arts and Sciences, with roughly 200 staff and a mission centered on fundamental and strategic neuroscience. Based in Amsterdam near Amsterdam UMC and Vrije Universiteit Amsterdam, it connects basic research with clinical collaborators and shared neuroimaging infrastructure. The Spinoza Centre for Neuroimaging, a collaboration with VU Amsterdam and Amsterdam UMC, offers 3T and 7T MRI capacity and welcomes industrial research programs; the Netherlands Brain Bank and Netherlands Sleep Registry support access to human tissue and longitudinal sleep data. Research is supported by KNAW and competitive funding from NWO, ZonMw, the European Commission, and philanthropic health foundations.
delivered to your inbox.