Unit profile
Research
Epilepsy is a devastating neurological disorder characterized by synchronous neuronal activity, which manifests as epileptic seizures. It usually develops after an initial insult, such as head trauma or stroke, which triggers the epileptogenic process. During epileptogenesis, the affected brain regions undergo multiple changes in connectivity and gene expression, and among the most affected genes are those responsible for network inhibition. Recent developments in gene editing technologies, after the discovery and application of the bacteria-derived CRISPR-Cas9 system, have opened new avenues for gene therapy. In particular, the possibility of developing a reversible and precise regulatory system for endogenous genes with high spatio-temporal control, overcomes several limitations of the classic gene therapy approaches. My group's research aims to apply an inducible gene modulation technology, based on guide RNAs and a catalytically inactive Cas9, targeting the expression of epileptogenic genes.
Collaborations the last five years
Profiles
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Marco Ledri
- Epilepsy Center - Principal investigator, Head of office, Associate researcher
- Experimental Epilepsy Group - Research project participant
- Molecular Neurophysiology and Epilepsy group - Research team manager
Person
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Gene therapy for epilepsy: An emerging, promising approach for a serious neurological disorder
Ledri, M. & Kokaia, M., 2026 Mar, In: Journal of Internal Medicine. 299, 3, p. 302-327 26 p.Research output: Contribution to journal › Review article › peer-review
Open Access -
Chemogenetics with PSAM4-GlyR decreases excitability and epileptiform activity in epileptic hippocampus
Gonzalez-Ramos, A., Berglind, F., Kudláček, J., Rocha, E. R., Melin, E., Sebastião, A. M., Valente, C. A., Ledri, M., Andersson, M. & Kokaia, M., 2025, In: Gene Therapy. 32, 2, p. 106-120Research output: Contribution to journal › Article › peer-review
Open Access -
Presynaptic nanoscale components of retrograde synaptic signaling
Barti, B., Dudok, B., Kenesei, K., Zöldi, M., Miczán, V., Balla, G. Y., Zala, D., Tasso, M., Sagheddu, C., Kisfali, M., Tóth, B., Ledri, M., Sylvester Vizi, E., Melis, M., Barna, L., Lenkei, Z., Soltész, I. & Katona, I., 2024 May, In: Science Advances. 10, 22, eado0077.Research output: Contribution to journal › Article › peer-review
Open Access
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Enhancing neuronal inhibition by cell and gene therapy as a novel treatment for Epilepsy
Gonzalez Ramos, A. (Research student), Kokaia, M. (Supervisor), Andersson, M. (Assistant supervisor) & Ledri, M. (Assistant supervisor)
2017/09/15 → 2022/04/08
Project: Dissertation
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On-demand RNA-guided gene therapy against epileptogenesis
Ledri, M. (PI), Di Maria, V. (Researcher), Raza, M. L. (Researcher) & Boulot, A. (Researcher)
2016/01/01 → 2019/12/31
Project: Research