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The Specific Topics of LOEWE-CGT:

- Reprogramming

The possibility to create pluripotent cells by reprogramming differentiated cells opens new perspectives for the generation of patient-specific stem cells for therapeutic use in practically every medical field. However, it is essential to improve our understanding of reprogramming, self-renewal and differentiation mechanisms in order to be able to achieve the safety standards necessary for clinical use. Manipulating epigenetic mechanisms to control gene expression and modification are of particular relevance to achieve efficient, virus-free reprogramming of adult cells.

- iPS

The production of pluripotent stem cells from patient-specific differentiated cells has for the first time made it possible to model disease-specific processes and their control mechanisms in cell culture. This research will allow investigation of the influence of disease-specific (coding) genetic alterations on disease-specific changes in cell development and differentiation. Similarly, targeted gene therapies can be tested and validated for their potential applicability as treatment procedures. Establishment of disease-specific induced pluripotent stem cell lines has opened completely new avenues to systematically search for innovative treatment strategies and is an especially ideal model to test combined cell and gene therapies.

- Stem Cell Homing

The function and differentiation of cells is essentially determined by the environment or niche. Therefore, a better understanding of the interaction of stem cells with their niches in healthy and diseased tissues is essential. This is also important for improved incorporation of therapeutically modified cells into the target tissues as well as for exploiting their "homing" mechanisms for endogenous repair.

- Stem Cell Transplatation and Cell Therapy

The use of allogeneic donors for cell therapy can result in dangerous immunological reactions after transplantation, especially when non-HLA identical donors are used. The understanding, circumvention and optimal manipulation of this immunological challenge are important requirements necessary for the therapeutic use of allogeneic cell therapies. In particular, the use of allogeneic cell therapies have a high potential in procedures aimed at the treatment of malignant diseases.

-Gene Therapy

Increased safety and efficiency of gene transfer vectors should be achieved through development of vectors with cell type specific surface proteins and/or tissue specific promoters. In addition to viral vectors, several new methods such as those involving RNA aptamers, miRNA inhibitors (Antagomirs) and microRNA mimetics have great therapeutic potential.

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