The field of gene ther­apy has been dili­gently mov­ing for­ward over the past sev­eral decades to bring po­ten­tially life-sav­ing treat­ments to pa­tients with ge­netic dis­eases. In ad­di­tion to two ap­proved adeno-as­so­ci­ated vi­ral (AAV) gene ther­a­pies, there are ...