Genomic medicine — the therapeutic use of cells and genetic materials — is at the center of a medical revolution. Approvals of genomic medicines are growing each year, fueled by new precision medicine platforms that target diseases without current effective treatments. Viral vectors, primarily adeno-associated virus (AAV), efficiently treat some diseases caused by monogenic mutations. Gene-modified cell therapies, notably CAR-T cell therapies, successfully treat some late-stage blood cancers but face obstacles for use with solid tumors.
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