some genes and thus leave a typical trace. The scientists use artificial intelligence to recognise this gene expression signature from eleven genes when blood stem cells are treated with the gene therapy [...] 2021 When the causes of serious diseases are based on a defective gene, medicine relies on gene therapy. In this strategy, defect-free genes are introduced into the body with the help of certain viruses. [...] can already be treated today. However, the viral vectors used as gene shuttles work in different ways. Some simply deliver the intact gene into the cell. The others belong to the retroviruses and incorporate

Microsurgery of the genome Traditional gene therapy attempts to replace the function of a defective gene, for example by introducing a correct version of the gene into the cell. "With genome editing, on [...] on this. The gene therapy experts from the Institute of Experimental Haematology at Hannover Medical School (MHH), together with five other experts, have summarised the current state of gene therapy in a [...] enables gene repair," adds Professor Schambach. "The same technique can also be used to improve the activity of cell therapeutics, such as those used in oncology, or to specifically switch off genes in order

already available gene therapy only helps certain patients who do not form antibodies against the viral vector, which acts as a gene carrier to introduce an intact copy of the FVIII gene into the body. Dr [...] amounts to around 60.000 euros. Established gene therapy does not help everyone Up to now, haemophilia A has been treated with the already established AAV gene therapy. Here, the protein envelope of the [...] the FVIII gene. As a viral vector, AAV introduces the FVIII copy into liver cells, which can then produce the corresponding protein - clotting factor VIII. AAV vectors are often used as gene shuttles because

single gene - also in the liver. These include the blood coagulation disorders haemophilia A or B or the metabolic disease phenylketonuria. Gene therapies could help here, in which intact genes are transported [...] severe muscle weakness and atrophy. In order to deliver the therapeutic genes to the target, so-called viral vectors are used as gene taxis. Among the best-known representatives are adeno-associated viruses [...] scientific journal Hepatology. Not all gene taxis reach their target Although AAV vectors are derived from viruses, they serve exclusively as a means of transport in gene therapy. With the viral envelope, the

tiny faulty building block in the mutated HFE gene. In technical jargon, the procedure is called base editing. The special feature of this gene repair: the gene scissors were used in such a way that they [...] haemochromatosis gene HFE, which is located on chromosome 6," says Professor Ott. It only occurs in people who have inherited this defect from both parents, i.e. who do not have a "healthy" gene to compensate [...] They use the body's own repair mechanisms to repair the defective HFE gene. With the help of CRISPR/Cas technology, known as "gene scissors", and an accompanying biotechnological tool, they have specifically

08.2022 Pathogenic variants in BRCA1/2 genes are associated with an increased risk of breast and ovarian cancer in adults and variants in mismatch repair (MMR) genes increase the risk of gastrointestinal [...] variants in BRCA1/2 and the MMR genes may also contribute to the risk of cancer in individuals below age 18 years. "Children and adolescents with BRCA1/2 or MMR gene variants were mainly affected by brain [...] healthy individuals for BRCA1/2 and MMR gene variants only from adulthood onwards, should such a variant be known in the family." However, detection of these gene changes may be of immediate importance

as a gene taxi, is intended to deliver the therapeutic gene precisely to the inner and outer cells of the cochlea in the inner ear by microinjection. "Since MYO7A is a very large gene, we need a gene taxi [...] balance. Responsible for this is a mutation in a gene called MYO7A, which Professor Schambach and his team are focusing on. If a person inherits the damaged gene from both father and mother, no healthy variant [...] 17. November 2022 Protecting children and adults from hereditary deafness with the help of gene therapy is one of the goals of Professor Dr. Dr. Axel Schambach, head of the Institute for Experimental

special gene scissor that only cuts the viral RNA genetic information, but leaves human RNA intact, which acts as a construction manual for certain proteins to convert the information of our genes," explains [...] tract, the blueprint is packaged in special gene shuttles and can be easily inhaled via a spray, for example. In the lungs, the body itself assembles the gene scissors, which are programmed to cut the viral [...] second aims to destroy the genetic material of the SARS-CoV-2 coronavirus with the help of so-called gene scissors. Fighting all hepatitis E strains with antibodies The hepatitis E virus (HEV) belongs to

TALEN or CRISPR/Cas9 methods, gene constructs containing therapeutic genes can be introduced into the cells to produce "suprafunctional" cells. Reporter or selection genes under the control of appropriate [...] caspase 9 suicide gene ( iCASP9 ) into the AAVS1 safe harbor locus efficiently indeed triggers cell apoptosis in vitro and in vivo , but only the integration of two copies of the suicide gene prevents the emergence [...] c mutations via "gene editing" Many human genetic diseases are caused by point mutations in the genome. One approach for the correction of disease-specific mutations in targeted gene therapy is using

02.08.2022 Gene therapies aim to cure severe, barely treatable monogentic diseases caused by a defect in a single gene. Medical hopes are correspondingly high. Some gene therapies have already been approved [...] so-called viral vectors, therapeutic genes are transported directly into the cell as a "drug". The best-known representatives of these vectors, colloquially known as gene taxis, are the so-called adeno-associated [...] the running and then tested how strongly they were intercepted by the human immune system." Not all gene taxis reach their target Not all vectors that target specific body cells get there. "About 70 per