Breakthroughs and challenges in gene therapy

Gene therapy aims to treat diseases by replacing, inactivating, or introducing genes into patients through a vector — either inside the body (in vivo) or outside of the body (ex vivo). Vectors typically used include nonintegrating viruses such as adeno-associated viruses (AAVs), integrating viruses such as lentiviruses, and non-viral vectors such as lipid nanoparticles (LNPs).

Some therapies are considered both cell and gene therapies. These therapies work by altering genes in specific types of cells and inserting them into the body. Therapies are being developed to target areas such as various types of cancer, and autoimmune and rare diseases.

This gene therapy feature shares the latest news and tech developments in cell therapy, RNA therapeutics, and cell line development.