Gene therapy is currently approved by the U.S. Food and Drug Administration (FDA) for only a few diseases—Leber congenital amaurosis , retinal dystrophy, beta thalassemia, hemophilia, and spinal muscular atrophy  are examples—but many more gene therapies are in development and clinical testing. Specific DNA sequences can now be targeted with clustered regularly interspaced short palindromic repeats (CRISPR)-associated nucleases to inactivate genes or to correct a pathogenic mutation back to the reference sequence. This technique has already demonstrated durable benefits for patients with hemophilia.

Cell-based therapy is now beginning to provide vehicles for the delivery of cells engineered to address a patient’s particular chimeric antigen receptor (CAR). CAR-T cell therapy is FDA-approved for conditions such as nonHodgkin lymphoma, acute lymphoblastic leukemia, multiple myeloma, melanoma, and prostate cancer, and it is being actively tested in other conditions. 

Although effective in many cases, cost and severe side effects, especially the so-called cytokine release syndrome, currently limit its widespread use.Regenerative medicine to help heal injured or diseased organs, by the administration of cells modified to meet a patient’s needs, is in its infancy. However, cultured chondrocytes are now FDA-approved to repair cartilaginous defects of the femoral condyle and the knee.

Immune checkpoint inhibitors have revolutionized the approach to treating cancer, initially melanoma, but now also a wide range of malignancies, with future potential efficacy for the treatment of refractory infectious diseases, autoimmune diseases, and serious allergic disorders.Messenger RNA (mRNA) vaccines, which deliver antigen-encoding mRNA into immune cells, where they stimulate an adaptive immune response, have revolutionized the approach to preventing severe acute respiratory syndrome coronavirus-2 and are increasingly being applied to other infections.

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