Regenerative Medicine
Cell therapy
Cell therapy is an approach that relies on the use of cells as therapeutics. For replacement therapy, the stem or progenitor cells can be obtained from the patient or a donor. These cells can generate a limited number of cell types.
Another option is to use pluripotent stem cells (PSC), which can generate any cell type of the body. Through increasingly well-defined differentiation protocols, multiple cell types of different organs can now be formed in the laboratory. At LUMC, human PSCs are central to many research groups intending to use the cells in therapy as Advanced Therapy Medicinal Products (ATMPs). In addition, we are in the process of developing protocols to optimize the isolation of islets of Langerhans and hepatocytes for the treatment of diabetes and acute (or chronic) liver failure.
…Cell therapy
Cell therapy is an approach that relies on the use of cells as therapeutics. For replacement therapy, the stem or progenitor cells can be obtained from the patient or a donor. These cells can generate a limited number of cell types.
Another option is to use pluripotent stem cells (PSC), which can generate any cell type of the body. Through increasingly well-defined differentiation protocols, multiple cell types of different organs can now be formed in the laboratory. At LUMC, human PSCs are central to many research groups intending to use the cells in therapy as Advanced Therapy Medicinal Products (ATMPs). In addition, we are in the process of developing protocols to optimize the isolation of islets of Langerhans and hepatocytes for the treatment of diabetes and acute (or chronic) liver failure.
Gene therapy
Gene therapy aims to repair genetic defects to restore a dysfunctional or missing protein in a patient’s cells or endow cells with novel functions (such as avoidance of immune rejection after transplantation or introducing tolerance to ischemic-reperfusion injury). All these applications are being developed and implemented at LUMC by researchers in the CTO Theme. Therapeutic genetic material (DNA or RNA) is transferred to the target cells in various ways like lentivirus, adenovirus, AAV, or non-viral delivery systems. As a result, the patient cells can produce the therapeutic protein specific to the disease. LUMC is at the forefront of the development of several gene therapies, such as carrying out the first clinical trial worldwide for RAG1 SCID, a genetic disorder that severely compromises the immune system. In addition, researchers of this theme are developing new technology based on CRISPR-Cas gene editing systems that restore the function of the endogenous affected gene.
Gene therapy projects
Related research facilities
Contact
Email: RegMedTheme@lumc.nl