The mission of the GRTC is to develop innovative, personalized, and broadly applicable Gene and RNA therapy approaches that are safe and affordable. By initiating and executing clinical gene and RNA therapy studies, we aspire to offer new perspectives to patients with rare diseases and target common diseases.
The unique combination of excellent basic, clinical and translational expertise at the Gene and RNA Therapy Center in Tübingen, along with the close collaborations with the MPI of Developmental Biology in Tübingen on the bioengineering of CRISPR/Cas9 and Base-/Prime-editors, the MPI of Biochemistry in Martinsried/Munich on Sendai based Virotherapy, and the biotech company CureVac on mRNA production for gene therapy, allows the development of safe, precise and affordable Gene and RNA therapy and cell therapy products for clinical use.
The existence of a Center for Rare Diseases and several International Registries for Rare Diseases at the Tübingen University working closely with the Institute of Medical Genetics and Applied Genomics, Deutsches Zentrum für Neurodegenerative Erkrankungen (DZNE), Comprehensive Cancer Center Tübingen-Stuttgart (CCC-TS), National Center of Tumor Diseases (NCT) as well as the Tübingen Early Clinical Trials Unit (ECTU) enables the identification of novel targets for Gene and RNA therapy and, importantly, allows high-volume recruitment of patients for both academic investigator-initiated (IITs) as well as Pharma trials. This makes Tübingen a highly attractive place for Gene and RNA therapy trials.
Nucleic acids (DNA and RNA) carry the code for life, providing a major platform to regulate health and disease. In fact, the journey of discovery of nucleic acids functions in health and disease started in Tübingen in 1869, when Friedrich Miescher identified a new substance called ‘nuclein’ in the nuclei of immune cells. This discovery ignited a revolution in life sciences and more recently in medicine, offering major innovations in diagnostics and therapies. Gene (DNA) and RNA therapy provides treatment options for disorders traditionally considered ‘undruggable’ and therefore is expected to extend the scale of treatable diseases dramatically, resulting in a medical revolution similar to that of monoclonal antibodies in the past 40 years. Gene and RNA therapy also offers fast-evolving, advanced tools to treat common and rare diseases, showing rapid and groundbreaking progress and revolutionizing medicine already now and in the near future.
Prof. Dr. Julia Skokowa, MD, PhD
Founding Director, Gene and RNA Therapy Center
Head, Division of Translational Oncology
Department of Oncology, Hematology, Clinical Immunology and Rheumatology
University Hospital Tübingen
Address:
Otfried-Müller-Str. 10
72076 Tübingen
Phone number: +49 7071 29-82168
Fax number: +49 7071 29-25161
E-mail address: Julia.Skokowa@med.uni-tuebingen.de
Dr. Olga Klimenkova
Scientific Coordinator, Gene and RNA Therapy Center
Department of Oncology, Hematology, Clinical Immunology and Rheumatology
University Hospital Tübingen
Address:
Otfried-Müller-Str. 10
72076 Tübingen
Phone number: +49 7071 29-86013
E-mail address: olga.klimenkova@med.uni-tuebingen.de
Coordinator / Director: Prof. Dr. Julia Skokowa, MD, PhD
External Advisory Board - Advising the Board of Directors
The board is composed of the spokespersons of the 4 areas:
The election of the Board of Directors and the Management Committee is held every 5 years.
Matthew Porteus (Chair), Professor, Department of Pediatrics- Stem Cell Transplantation, Stanford Medical School, USA.
Annemieke Aartsma-Rus, Professor of Translational Genetics, Department of Human Genetics at Leiden University, The Netherlands.
Alberto Auricchio, Associate Professor of Medical Genetics at the University of Naples and Director of Research at the Telethon Institute of Genetics and Medicine in Naples, Italy.
Christopher Baum, Professor, Chief Translational Research Officer / Chairman of the Board of the Berlin Institute of Health, Charite, Berlin, Germany.
Hildegard Bühning, Professor of Infection Biology and Gene Transfer and Deputy Director of the Institute of Experimental Hematology, Hannover Medical School, Germany. President of the European Society for Gene and Cell Therapy and Scientific Secretary of the German Society for Gene Therapy.
Christine Engeland, Professor of Experimental Virology at Witten/Herdecke University, Germany and Head of the Research Group "Mechanisms of Oncolytic Immunotherapy" at Heidelberg University Hospital, National Center for Tumor Diseases and German Cancer Research Center in Heidelberg.
Boris Fehse, Professor, Head of the Cell and Gene Therapy Research Unit at the Clinic for Stem Cell Transplantation, UKE Hamburg. Scientific Advisory Board (past president) of the Society for Gene Therapy.
Anastasia Khvorova, Professor at the RNA Therapeutic Institute, UMass Medical School, USA.
Martin Meier, PhD, Vice President, Research, Alnylam Pharmaceuticals.
Luigi Naldini, Professor of Cell and Tissue Biology and Gene and Cell Therapy at San Raffaele University School of Medicine. Director of the San Raffaele Telethon Institute for Gene Therapy, Milan, Italy.
Dirk Nettelbeck, PD Dr., Project Leader in the Clinical Cooperation Unit Virotherapy at DKFZ, National Center for Tumor Diseases and German Cancer Research Center in Heidelberg.
Pediatrics
Autosomal recessive and early onset ataxias
Friedreich's ataxia
Glioblastoma
Hearing impairment
Hereditary spastic paraplegia
Inherited Leukemias
Ba-Wü HemPred Board für Leukämie-Prädisposition
Inherited Neutropenia
Neuroendokrine Tumore (NET)
Spinocerebellar Ataxia
Sporadic cerebellar ataxia and Multiple system atrophy
Angeborene Neutropenie
Hämophilie
Hereditäre Spastische Spinalparalyse
Neuroendokrine Tumore (NET)
Schwerhörigkeit und Taubheit
Shwachman Diamond Syndrom (SDS)
