Information on Gene and Cell-Based Therapies for Patients

Gene- and cell-based therapies (GCT) offer promising new approaches for the treatment of previously incurable diseases and can support regenerative processes. They open up entirely new perspectives for patients with severe and rare conditions as well as for those affected by common diseases such as diabetes, Parkinson’s disease and autoimmune disorders.

These two approaches are often combined. For instance, when cells are first genetically modified, then multiplied, and finally reintroduced into the body.

Cell therapy uses healthy cells to repair damaged tissue, correct a malfunctioning immune system, or fight tumors.

• Stem cell therapy: Utilizes healthy human stem cells, which can develop into different cell types, to replace damaged tissue (e.g., bone marrow transplantation for blood cancer).
• Immune cell therapy: Uses, for example, the patient's own T cells. These are genetically modified or activated to attack cancer cells.

In gene therapy, DNA or RNA is introduced into cells, removed, or modified to treat diseases. There are different approaches:

• Insertion of a functional gene: A healthy gene replaces a defective or missing gene (e.g., in spinal muscular atrophy or hemophilia B).
• Correction or disabling a defective gene: Technologies such as CRISPR/Cas9 (the so-called gene scissors), TALENs, or zinc finger nucleases can be used to correct genetic mutations (e.g., for inherited blindness, sickle cell anemia, or Duchenne muscular dystrophy).

Further information is currently being developed and published gradually by a subgroup of Topic VIII "Interaction with society".