Adeno-associated virus serotype 1-based gene therapy for FTD caused by GRN mutations.
Christian HindererRod MillerCecilia DyerJulia JohanssonPeter BellElizabeth BuzaJames M WilsonPublished in: Annals of clinical and translational neurology (2020)
These findings support the development of AAV1-based gene therapy for frontotemporal dementia caused by progranulin deficiency.