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Progranulin AAV gene therapy for frontotemporal dementia: translational studies and phase 1/2 trial interim results

Interim results from the low-dose cohort of the PROCLAIM trial show that AAV-based delivery of the progranulin gene in patients with frontotemporal dementia was safe and was associated with increased CSF progranulin.

Jeffrey Sevigny
Olga Uspenskaya
Franz Hefti
ResearchOpen Access14 May 2024
Nature Medicine

Volume: 30, P: 1406-1415

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Progranulin AAV gene therapy for frontotemporal dementia: translational studies and phase 1/2 trial interim results

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