When Immune Suppression Goes Wrong

Many clinical in vivo gene therapies for genetic disease, ranging from early-stage trials to approved medicines, utilize adeno-associated viral (AAV) vectors. Nonetheless, immunogenicity remains a major obstacle. Humoral and cellular immune responses against the vector’s capsid or the transgene product can lead to a loss of therapy, prevent re-administration of the vector, and cause immune toxicities that are potentially harmful to the...