[저자] Jeffrey Sevigny, Olga Uspenskaya, Laura Dean Heckman, Li Chin Wong, Daniel A. Hatch, Ambika Tewari, Rik Vandenberghe, David J. Irwin, Dario Saracino, Isabelle Le Ber, Rebekah Ahmed, Jonathan D. Rohrer, Adam L. Boxer, Sebastian Boland, Patricia Sheehan, Alissa Brandes, Suzanne R. Burstein, Benjamin M. Shykind, Sitharthan Kamalakaran, Carter W. Daniels, E. David Litwack, Erin Mahoney, Jenny Velaga, Ilan McNamara, Patricia Sondergaard, Syed A. Sajjad, Yvonne M. Kobayashi, Asa Abeliovich, Franz Hefti
[Category] update2024,
[Article Type] Article
[Source] PMC
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.
All Keywords
【저자키워드】 Target validation, Neurodegenerative diseases,