Gene- and Cell-based therapies (GCT) offer promising approaches to treating previously incurable diseases and can support regenerative processes. Gene-based therapies use nucleic acids, i.e. DNA or RNA, which can be used to regulate or add biological functions. Cell therapies generate and/or transfer cells into the organism for therapeutic purposes. These two approaches can also be combined, e.g. using genetically modified cells.
Gene and cell-based therapies include the following procedures (not definitive):
The following procedures, however, are not included:
GCTs have an effect that goes beyond purely symptomatic treatment, as they target the causes of disease specifically - with the prospect of cure and regeneration. Their range of applications extends from rare genetic diseases to more common and complex diseases, some of which still have no effective treatments. Despite existing research questions regarding safety and efficacy, millions of patients in Germany alone could benefit from GCT.