Gene and RNA Therapy Center (GRTC)

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Address: Otfried-Müller-Straße 10
72076 Tübingen

Founding Director

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Prof. Dr. Julia Skokowa

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Scientific coordinator

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Dr. Olga Klimenkova

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AG Cheng

Dr. Fubo Cheng

Institute of Medical Genetics and Applied Genomics

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Prof. Dr. med. Olaf Rieß

Director of the Institute of Medical Genetics and Applied Genomics

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Dystonia and Parkinson’s disease research group

Evidence from patients with Parkinson’s disease (PD) and alpha-synuclein (SNCA) transgenic animal model support the idea that increased SNCA protein is a substantial risk factor of PD pathogenesis. Thus, targeted modifying the expression of SNCA as gene therapies for PD is one of the research interests of our group. Primary dystonia is the third most common movement disorders and lacks of efficient treatment so far. Finding new potential targets for gene therapy is another research interest of our research group. By combining multiple disease model systems with CRISPR/Cas9 based gene editing and gapmer ASOs, we are trying to find out new genetic therapies for these diseases.

List of GRT projects

CRISPR/Cas9 gene editing as treatment for Parkinson’s disease and primary dystonia
ASO therapy for Parkinson’s disease and primary dystonia

GRT expertise

Animal models for Parkinson’s disease and primary dystonia
CRISPR/Cas9 based gene editing for Parkinson’s disease and primary dystonia
ASO therapy for Parkinson’s disease and primary dystonia

Main GRT methods applied in the lab

CRISPR/Cas9 gene editing methods
Experiments with animal models
Next generation sequencing (NGS) based methods

Ongoing and requested funding

DFG: Role of the Dystonia type 6 gene product THAP1 in gene regulation and in neurological diseases (https://gepris.dfg.de/gepris/projekt/505858170).
DFG: Targeted modifying the human SNCA expression as gene therapy for Parkinson’s disease. (Requested)

Publications

Cheng F, Zheng W, Liu C, Barbuti P, Yu-Taeger L, Casadei N, Huebener-Schmid J, Admard J, Boldt K, Junger K, Ueffing M, Houlden H, Sharma M, Krüger R, Grundmann-Hauser K, Ott T, Riess O. Intronic enhancers of the human SNCA gene predominantly regulate its expression in brain in vivo. Sci. Adv., 2022; 8(47), eabq6324. DOI: 10.1126/sciadv.abq6324.
Cheng F, Zheng W, Barbuti PA, Bonsi P, Liu C, Casadei N, Ponterio G, Meringolo G, Admard J, Dording CM, Yu-Taeger Y, Nguyen HP, Grundmann-Hauser, Ott T, Houlden H, Pisani A, Krueger R, Riess O. DYT6 mutated THAP1 is a cell type dependent regulator of the SP1 family. Brain. 2022; Nov 21;145(11):3968-3984. doi: 10.1093/brain/awac001.
Cheng FB, Feng JC, Ma LY, Miao J, Ott T, Wan XH, Grundmann K. Combined occurrence of a novel TOR1A and a THAP1 mutation in primary dystonia. Mov Disord. 2014 Jul;29(8):1079-83.

List of all publications

Relevant links

Institute of Medical Genetics and Applied Genomics