Abstract: Described herein are methods for treating a subject having or at risk of developing a neurocognitive disorder, such as frontotemporal lobar degeneration or neuronal ceroid lipofuscinosis, by administering cells that contain a transgene encoding a progranulin (PGRN) or a granulin (GRN) or cells that express the PGRN or the GRN to the subject. Also disclosed are compositions comprising cells containing the transgene encoding the PGRN or the GRN.
Type:
Application
Filed:
January 31, 2020
Publication date:
April 14, 2022
Applicants:
AvroBio, Inc., AvroBio, Inc.
Inventors:
Chris MASON, Oliver COOPER, Mark DEANDRADE, Robert PLASSCHAERT, Nico Peter (Niek) VAN TIL