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, Senior 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.,Head of the research group Oncolytic Adenoviruses in the Clinical Cooperation Unit Virotherapy at the German Cancer Research Center (DKFZ) 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)
Thomas Gasser, Hertie Institute for Clinical Brain Research ...
Learn moreThe next Annual Meeting of the German Society for Gene Therapy.
Learn moreGene therapy is the use of genetic material in the treatment or prevention of disease. Gene therapy can be used to reduce the causes of a disease or increase the ability of the body to fight such diseases.
Gene therapy is the treatment of disorders by fixing faulty gene functions that would otherwise cause disease and disorders, instead of using drugs or surgery to treat those disorders. In short, the traditional focus on treating disease and disorders turns toward the more optimistic vision of stopping diseases and disorders.
Gene therapies are still new and so there is still some uncertainty around their efficacy and long-term effects. Therefore, safety is paramount. This is the reason why there is extensive basic research, pre-clinical and clinical trails to make sure, like traditional therapies, it is as safe as possible.
Some people say that any interventions on DNA is dangerous and unnatural. Some uses – such as enhancements – might be considered problematic in this way. However, focusing on therapy suggests that it is not more intrinsically unnatural then modern medicine itself.
DNA-based gene therapies should create permanent changes, thus valuable if beneficial changes; while RNA therapies should not be permanent, thus treatment can be modified or halted if necessary.
Your health data, especially your genetic data is very sensitive material, which can be very impactful in terms of insurance, employment, and future health and family decisions. Therefore, it is a priority to ensure its security and to ensure your privacy. Adherence to the General Data Protection Regulation (GDPR), your genetic data is treated as a special category of importance.
Incidental findings are results which were not related to the intention of the genetic (or other) test and research undertaken. Sometimes they may have important clinical implications. Other times, they may be risk factors which may be of uncertain significance (e.g. a possible increased chance of a later-life onset disorder). This would require careful assessment of risk and discussion with you would be undertaken should this arise.
Something called informed consent is a central value in all modern medicine, especially genome/RNA medicine. An informed consent is not just asking – but asking in a context where you have all the relevant information about what is involved (risks and benefits), and that you are supported in your decision-making, so that you can make the right decision for you.
In the context with your GP, treatment is generally with established methods, with well-known risk/benefit balances and a relative confidence that there will be a positive, intended therapeutic outcome. In the context of therapeutic research, it is still to be determined what the therapeutic outcome will be, how good it will be, what the balance of risks and benefits will be. Therefore, the therapeutic research context requires a more stringent form of informed consent, with greater protections for you.
The interventions that take place at the GRTC are not passed to your children. Some genetic interventions are called somatic which involves interventions on genes in the body’s cells where needed – e.g. eye, blood. This does not affect germ cells that produce the eggs and sperm and so does not affect the patient’s children or descendants. If they did, they would be called germline interventions.
As your genetics is shared with your family members, so too is information that may arise regarding genetic abnormalities. However, confidentiality is a central principle in western medicine, and you have control over who will learn about such information. Genetic councilling is important in such situations so that you are helped to discuss and reflect, in a non-judgemental context, on what you should do.
