Reducing the mortality rate of patients after Aortic-Valve Implantation by Anti-Fibrotic Treatment
A subpopulation of patients with a very high risk to die in only one year after an aortic valve implantation (AVI) was identified. A novel, anti-fibrotic treatment was developed, which adresses the most important factor for the survival rate of patients and is being tested in a phase 3 clinical trial.
Pan-Coronavirus Therapy – a Cas13-based antiviral approach
Scientists from the University of Göttingen developed a strategy for a therapy which will work against all Coronaviruses known to infect humans. The antiviral effect is based on a Cas13-guideRNA System, which directly and specifically targets the genome of the Coronavirus. A precise acute treatment in the early phase of the infection is possible, but importantly also an immunity against an infection lasting for several month will be impartet to cells expressing the Cas13-guideRNA System.
Dopamine producing organoids for therapy of Parkinson’s Disease
Researchers at the University Medical Center in Göttingen, Germany, have developed a method to produce special brain organoids called „dopamine-producing neuronal organoids“ (d-NOs). These organoids have been shown to release dopamine at levels that are effective for biological processes. This method is ideal for the production of an advanced therapy medicinal tissue-engineered product (ATMP-TEP) for clinical use in Parkinson’s disease. Furthermore, the invention provides a method for producing d-NOs for use in disease modelling, drug screening and drug development.
Novel bacterial Cry proteins for treating parasitic nematodes in humans and animals
Gastrointestinal nematode (GIN) infections are a significant issue, particularly in animals that graze. They can have a severe impact on cattle health. GIN primarily affect the host animal’s nutritional status by causing tissue lesions, inducing inflammatory reactions, or by feeding directly on the animal’s blood. Helminth infections are predicted to cost the European ruminant livestock industry € 1.8 billion annually. They represent a significant health cost to dairy farmers, ranking second only to mastitis.
Activity-improved human Asparaginase for Cancer therapy
Amino acid depletion therapy offers a promising approach to cancer treatment. It makes use of the differences between healthy and cancerous cells when processing nutrients. Some enzymes kill cancer cells by removing amino acids from the blood. Asparaginase (ASNase) is an FDA-approved enzyme used to treat acute lymphoblastic leukemia (ALL). ALL is the most common cancer in children. ASNase turns asparagine into aspartic acid and ammonia. This reduces the amount of asparagine in the blood and stops leukemic cells getting the asparagine they need to make DNA, RNA and protein, selectively which kills them but not healthy cells. Today’s clinics use enzymes from bacteria. They cause many different side effects, but the main one is hypersensitivity (i.e. allergic reactions due to antigenicity).
Tissue-engineered Human Heart Muscle from stem cells for Screening and for Heart Repair
Heart tissue engineering using stem cells is a recently developed technique to construct a three dimensional cell/tissue structure from progenitor cells. Those engineered tissues can be either used for in vitro screenings (e.g. drug or toxicology screenings) or as a therapeutic tool to replace damaged or diseased tissue (regenerative medicine). Scientists at the University of Göttingen developed two new and fully defined methods for serum-free production of human engineered heart muscles (EHM) either from pre-differentiated cardiomyocytes or directly from undifferentiated stem cells. This will uniquely allow cardiological screening by measurement of muscular contraction (inotropic effects) on human tissue for the first time.
Bio-engineered neuronal organoids from human stem cells
In vitro 3D modeling of neural tissues represents a powerful tissue bioengineering tool permitting the study of complex neuronal cell systems. Despite the advances in human neuronal organoid generation, there still exist numerous deficiencies limiting the utility of existing neuronal organoids. Scientists at the University Medical Center of University Göttingen developed a robust, reproducible and fully defined method for serum-free production of human bio-engineered neuronal Organoids (BENOs) from stem cells having functional neuronal network activity.
Pharmacologically controlled vector for CNS gene therapies
Current gene therapy is irreversible and does not allow controlled transgene expression in case of side effects. The invention is a pharmacologically controlled one-vector expression system of a therapeutic factor (i.e. GDNF) with zero background expression, based on mifepristone (Mfp)-Gene Switch system arranging two cassettes.
Synthesis of prodrugs with cleavable linker for targeted tumor therapy (ADC)
Antibody tumor therapies have provided great therapeutic benefit to patients with cancer, autoimmune diseases and other serious medical conditions. However, many antibodies lack sufficient intrinsic anti-tumor activity to be used as first-line therapeutics. Scientists at the University of Göttingen, Germany developed a new and improved synthesis of potent prodrugs (payloads), which can be easily coupled to antibodies (ADC) for a selective antibody tumor therapy.
Targeted Demethylating Gene Therapy of Fibrosis
All-in-one construct dCas9-TET3CD-(target gene)-sgRNA for targeted fibrosis therapy through demethylation of the genes RASAL1, LRFN2, KLOTHO (i.e. in heart, kidney, liver, lung, cancer). Normal re-expression of these genes has been achieved successfully in vitro and in vivo as well as a reduction of fibrosis.