Gene Therapy

Gene Therapy or genetic modification is defined as the insertion of normal or genetically altered genes into cells using recombinant DNA technology to replace defective genes as part of the treatment of genetic disorders. It is considered a promising therapeutic option for many currently incurable diseases. Gene Therapy can be achieved by the genetic engineering of viral or non-viral vectors expressing therapeutic genes or by the implantation of genetically altered cells or tissues. Gene therapy is a young discipline, and a first clinical trial was performed in 1990 with more than 4000 clinical studies performed since then. Although there were great success stories in the early days, progress in the field towards clinical application was delayed, resulting from vector induced toxicity and tumorigenesis. However, recent advancements in vector development with enhanced safety profile and targeting capacity are very encouraging and will help to further develop gene therapy as a therapeutic option for many serious diseases. Moreover, the development of novel gene modifying techniques such as Crispr/Cas9 will further contribute to the development of safe gene therapies. 

The REMEDI Gene Therapy programme

Gene Therapy research at REMEDI focusses on several areas that are fundamental to the development of advanced therapies for major unmet health challenges such as cardiovascular disease, diabetes, eye disease, bone and joint disease, muscle wasting, kidney disease, cancer, lung disease and sepsis. REMEDI researchers use gene therapy to (i) genetically alter mesenchymal stem/stromal cells for enhanced immunomodulation and survival to modulate ocular injury and inflammation; (ii) to enhance the angiogenic and wound healing properties of the modified MSC; (iii) to reverse disease-induced cell dysfunction and (iv) to generate induced pluripotent stem cells (iPS) for cardiovascular, neural and musculo-skeletal disorders. REMEDI gene therapy researchers use a wide variety of gene vector types and provide scientific oversight for key methodologies such as viral and non-viral gene vector development and gene transduction.  

Relevant Research Groups