US 12,460,262 B2
Methods of detecting, preventing, reversing, and treating neurological diseases
Mark S. Sands, St. Louis, MO (US); and Bruno Benitez Viloria, St. Louis, MO (US)
Assigned to Washington University, St. Louis, MO (US)
Appl. No. 17/311,970
Filed by Washington University, St. Louis, MO (US)
PCT Filed Dec. 5, 2019, PCT No. PCT/US2019/064683
§ 371(c)(1), (2) Date Jun. 8, 2021,
PCT Pub. No. WO2020/118056, PCT Pub. Date Jun. 11, 2020.
Claims priority of provisional application 62/775,626, filed on Dec. 5, 2018.
Prior Publication US 2022/0025461 A1, Jan. 27, 2022
Int. Cl. A61P 25/28 (2006.01); A61K 48/00 (2006.01); A61P 25/16 (2006.01); C12Q 1/6883 (2018.01)
CPC C12Q 1/6883 (2013.01) [A61K 48/00 (2013.01); A61P 25/16 (2018.01); A61P 25/28 (2018.01); C12Q 2600/106 (2013.01); C12Q 2600/156 (2013.01); G01N 2800/28 (2013.01)] 8 Claims
 
1. A method of preventing, treating, reversing, or delaying Alzheimer's disease dementia of the Alzheimer's type in a subject in need thereof, comprising:
administering a therapeutically effective amount of an autophagy-lysosomal pathway agent comprising a gene for gene therapy (GT) to the subject,
wherein the subject has been determined to have or be at risk for Alzheimer's disease or dementia of the Alzheimer's type by virtue of detecting that the subject is heterozygous for a loss-of-function variant in lysosomal gene PPT1, and wherein the gene for gene therapy is a wild-type copy of the lysosomal gene.