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

the European Society of Gene and Cell Therapy (ESGCT) 4th Annual Congress of the German Society for Stem Cell Research (GSZ) 16th Annual meeting of the German Society for Gene Therapy (DG-GT) 2nd Meeting [...] combined meeting of the „European Society of Gene and Cell Therapy (ESGCT)“, the “German Society for Stem Cell Research (GSZ)“, the „German Society for Gene Therapy (DG-GT)“ and the „International Society [...] REBIRTH will take place on November 20-25, 2009 in Hannover. The main foci of the meeting include gene therapy, cell therapy, stem cell biology and regenerative medicine. Approx. 1,000 international p

Science Bio Informatics CRISPR/Cas9 gene editing in primary T cells Digital Fluorescence microscopy techniques, confocal microscopy and cell transfection Flow-cytometry Gene technology Gnotobiology and rederivation [...] capture of transcribed sequences [SCOTS] Proteome analysis, digital fluorescence microscopy Retroviral gene transfer Single cell immunophenotyping by ChipCytometry Software tools for imaging in infection S

the analysis of blood samples for the analysis of candidate genes of congenital neutropenias or for the analysis of the G-CSF receptor gene CSF3R in the context of our research projects. Please contact [...] leukemia development in blood cells of affected patients. The investigation of acquired mutations in the gene for the G-CSF receptor (CSF3R) in our laboratory has become an important standard diagnostic tool

mammalian cell culture; transfection; stable and transient gene expression; gene silencing (siRNA- or shRNA-mediated knock-down); CRISPR/Cas-mediated gene editing; cell toxicity assays Microscopy: epifluorescence

Leszek Lisowski „The future is here! The next generation of AAV vector as a foundation of cell and gene therapeutics of the future“ Associate Professor Lisowski received his PhD from Weill Cornell University [...] evaluation. In 2012, he was recruited by the Salk Institute for Biological Studies to oversee their Gene Transfer, Targeting and Therapeutic (GT3) facility. In 2015, he was recruited by the University of [...] purification, with special interest in clinical vector manufacturing. His team is leading a number of gene therapy programs that aim to develop novel advanced therapeutics for paediatric genetic disorders

complexity arises from regulation and does not correlate with the number of genes. The lab focuses on the regulation of gene expression and RNA biology. In terms of viruses we dissect the regulation of [...] causing various human diseases. Both areas follow our central idea: A deeper knowledge of both viral gene expression and the pathomechanisms of non-coding mutations will advance our understanding of cellular [...] medal: Noncoding mutations reveal new pathological mechanisms and insights into the regulation of gene expression. Wiley Interdiscip Rev RNA. 2021 Jan;12(1):e1616. Review. 2. Krooss S, Werwitzke S, Kopp

customized designer nucleases has been shown to be significantly more efficient than conventional gene targeting, but still typically depends on the introduction of additional genetic selection. This genome [...] “Stem Cell Reports” in January 2014. In this study, the authors demonstrate a selection-independent gene targeting without the typically required antibiotic selection. This optimized approach allows targeted [...] and robust expression and enables the introduction of mutations on nucleotide level as well as the gene correction of disease-specific mutations in patient-derived iPSCs. This method is not only of utmost

the considerable polymorphism of the genes underlying the HLA complex, more than 24,000 alleles can currently be differentiated at the transplantation-relevant HLA gene sites A, B, C, DRB1, DQB1 and DPB1 [...] transplantation and in patients prior to stem cell transplantation if a mismatch is present in one of the genes relevant for stem cell transplantation (HLA-A, B, C, DRB1, DQB1, DPB1). In patients on the kidney [...] available. A donor is considered identical if at least one high-resolution identity exists for the genes HLA-A, B, C, DRB1, DQB1. More than nine million registered stem cell donors are currently available

and gene regulation Flow of genetic information The cytoskeleton Microscopic techniques Membrane transport of small molecules Receptors and signal transduction Cell cycle and oncogenesis Human Gene finding [...] lymphoid organs Gene therapy : Stem cells, regulations of transgenes in somatic cells, embryonic and somatic cloning in mammals, Safety and efficiency in the use of viral vectors for gene therapy Microbiology [...] Scientific English 2.5 Radiation protection 2.7 Gene technological security 2.8 Introduction to ethics Ethical cases in medicine Ethical aspects of gene technology Scientific ethics 3. Other Courses 3