Modification of human genes in order to treat genetic diseases is referred to as gene therapy. Gene therapy is a medical procedure that involves inserting genetic material into a patients cells to repair or fix a malfunctioning gene in order to treat hereditary illnesses. Between 1989 and December 2018, over 2,900 clinical trials of gene therapies were conducted, with more than half of them in phase I. Since that time, many gene therapy based drugs became available, such as Zolgensma and Patisiran. Most of these approaches utilize viral vectors, such as adeno-associated viruses (AAVs), adenoviruses (AV) and lentiviruses (LV), for inserting or replacing transgenes in vivo or ex vivo'.
In 2023, nanoparticles that act similarly to viral vectors were created. These nanoparticles, called bioorthgonal engineered virus-like recombinant biosomes, display strong and rapid binding capabilities to LDL receptors on cell surfaces, allowing them to enter cells efficiently and deliver genes to specific target areas, such as tumor and arthritic tissues.
RNA interference-based agents, such as zilebesiran, contain siRNA which binds with mRNA of the target cells, modifying gene expression.