Project REVeyeVE - 06/02/2025

Targeted, virus-free gene therapy for the eye using degradable nanopropellers

Eye diseases that result in blindness in young people are primarily caused by genetic mutations. An interdisciplinary team of researchers from the Universities of Tübingen and Heidelberg is developing an innovative gene therapy method using biodegradable, magnetic nanopropellers. These innovative nanopropellers can effectively deliver intact genes into the affected cells, offering a potential solution for treating genetic disorders of this kind.

They are underestimated genetic control elements: it is known that changes in the genome can trigger diabetes. But now researchers at the University Hospital Ulm and the INSERM Cochin Institute in Paris have shown that a previously under-researched region of the genome also plays a crucial role in the development of this disease.

The summer reception hosted by BioRegio STERN Management GmbH has once again provided a fitting backdrop for the Science2Start award ceremony. Last Thursday, at Tübingen observatory, was the 15th time that scientists and start-up founders were celebrated for outstanding ideas that a panel of experts judged to have special economic potential.

How do tumors react to a certain therapeutic approach? Knowing this before the start of a therapy would be of enormous value for people suffering from cancer as well as for the doctors treating them. Researchers have now made this very observation possible for the CAR-T cell therapy. This allows us to individually investigate how exactly these tumor cells react to the planned therapy.

Applying and developing new technologies for DNA synthesis to pave the way for producing entire artificial genomes – that is the goal of a new interdisciplinary center, 'Center for Synthetic Genomics', that is being established at Heidelberg University, Karlsruhe Institute of Technology (KIT), and Johannes Gutenberg University Mainz (JGU).

Review - 09/02/2023

5th Gene Technology Report – a critical observation of a cutting-edge technology

The societal importance of genetic technologies was demonstrated during the coronavirus pandemic, when it was possible to rapidly develop suitable vaccines thanks to genetic engineering methods. As a result, the Fifth Gene Technology Report published in autumn 2021 reads like a validation of many years of work, as well as making it clear that the will to continue the detailed long-term monitoring is very much present.

With its "Proof of Concept" grants, the European Research Council ERC supports scientists in further developing the commercial potential of their research results. Nina Papavasiliou from the DKFZ is now receiving the prestigious grant for the second time: she wants to advance the development of a "molecular delivery service" that ensures that therapeutic genes reach the right address in the body in a targeted manner.

Today, genome editing is almost as easy as programming software. However, the generation of viral vectors as initial material is still associated with many expensive and error-prone handling procedures. Viruses are generated via complex biological processes that have to be optimised virus-specifically in order to produce high-quality therapeutics. A new method is needed that simplifies and optimises these processes.

AaviGen GmbH: Hope for heart failure - 14/07/2022

Gene therapy for weakened hearts

Treating a weak heart – for example after a heart attack – at the GP’s surgery with a single intravenous injection, without causing serious side effects: what sounds almost too good to be true is actually already under development. The Heidelberg-based biotech company AaviGen is working on a platform technology based on adeno-associated viruses that can help introduce therapeutic genes in a highly specific way into diseased heart muscle cells.

The nuclease Cas13b associated with the CRISPR gene scissors, which is an enzyme that degrades nucleic acids, has the potential to be used in the future in hereditary diseases to switch off unwanted genes. In the fight against infections, this nuclease is also being researched as an antiviral agent, as Cas13b can specifically intervene in the genetic material of viruses and render them harmless.

SolidCAR-T project - 15/03/2022

Modular ‘mini-factories’ for decentralised production of CAR T cells

Novel CAR T-cell therapies have proved to be promising therapeutic options for the treatment of acute leukaemias and lymphomas. Researchers from the Fraunhofer IPA in Stuttgart, the University Hospital Tübingen and the NMI in Reutlingen have joined forces in the SolidCAR-T project that aims to generate CAR T cells to combat solid tumours and produce these cells directly on site in the clinic using automated 'mini-factories'.

Dossier - 16/12/2021

Advanced therapy medicinal products: gene and cell therapies

Novel gene and cell therapies for treating incurable and hereditary diseases have raised high expectations. However, success has so far been limited to the long-established bone marrow transplants involving the administration of haematopoietic stem cells used to treat blood cancer. CAR T-cell therapies have recently emerged as a major new hope in cancer treatment.

Article - 29/01/2019

Targeted RNA editing with the body’s own enzyme activity

Completely new possibilities for research and gene therapy became available following the development of the CRISPR/Cas method for targeted modification of the genome. However, treatment with molecular scissors is not without risk as potential errors are stored in the genome forever. Scientists from Tübingen have developed an alternative method in which the intervention takes place at the RNA level using the body's own enzymes and is thus…

Article - 10/01/2019

Stocktaking and recommendations for action: the BBAW’s fourth gene technology report

In the new gene technology report, the interdisciplinary working group of the Berlin-Brandenburg Academy of Sciences (BBAW) takes stock of gene technology developments in Germany during the past few decades, and discusses the societal, legal and ethical challenges associated with these technologies in the future. The report is highly topical due to the controversy surrounding the ruling of the European Court of Justice on CRISPR/Cas9 genome…

Dossier - 28/08/2018

With molecular diagnostics to biomarker-based personalised therapy

Diagnosing suitable biomarkers is a prerequisite for tailoring personalised therapies to patient heterogeneity. Genetic tests and genome sequencing play a key role in these diagnoses. Up until now, personalised therapy has achieved the greatest success in the field of oncology. However, personalised treatments are also gaining in importance for treating other diseases.

Dossier - 14/06/2016

CRISPR/Cas – genome editing is becoming increasingly popular

The number of publications and patents that involve the CRISPR/Cas system has been increasing exponentially since the technique was first described a few years ago. The increase in funding for projects involving CRISPR/Cas also demonstrates how powerful this new method is. The targeted modification of genomes (also called gene or genome editing) using CRISPR/Cas is extraordinarily accurate and also has the potential to cure hereditary diseases.

Dossier - 10/11/2014

Cell and gene therapies from bench to bedside

While cell therapy has become standard treatment for a number of blood cancers, most cell and gene therapy approaches for the treatment of hereditary and metabolic diseases, neurodegenerative disorders and cancer are still in the experimental phases or early clinical trials. However, recent successes give rise to the hope that cell and gene therapies will in future make important contributions to previously incurable diseases.

Dossier - 01/04/2013

Retroviruses from infectious agent to therapeutic assistant

Viruses are infectious particles that use the machinery and metabolism of a host cell to replicate. The family of retroviruses is particularly known for its most notorious representative i. e. the human immunodeficiency virus HIV. However retroviruses are not only of interest for researchers looking for effective cures for viral infections their characteristic properties also make them promising laboratory and gene therapy tools.