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

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

J. Sevigny, O. Uspenskaya, et al.

Exciting breakthroughs in gene therapy with PR006 show promise in treating frontotemporal dementia caused by GRN mutations. This transformational research, led by a talented team of authors including Jeffrey Sevigny, Olga Uspenskaya, and others from Prevail Therapeutics, revealed encouraging safety and efficacy results, with all patients showing increases in crucial neuroprotective proteins. Join us on this journey to unlock new horizons in dementia treatment!... show more
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