US 12,275,764 B2
Methods of reducing FUS/TLS- or TDP-43-mediated neuronal cytotoxicity in amyotrophic lateral sclerosis (ALS) by UPF2
Greg Petsko, New York, NY (US); Dagmar Ringe, Cambridge, MA (US); and Shulin Ju, Beavercreek, OH (US)
Assigned to Brandeis University, Waltham, MA (US)
Filed by Brandeis University, Waltham, MA (US)
Filed on Apr. 15, 2022, as Appl. No. 17/721,715.
Application 17/721,715 is a continuation of application No. 16/739,561, filed on Jan. 10, 2020, granted, now 11,332,504.
Application 16/739,561 is a continuation of application No. 14/434,737, granted, now 10,533,038, issued on Jan. 14, 2020, previously published as PCT/US2013/063858, filed on Oct. 8, 2013.
Claims priority of provisional application 61/712,322, filed on Oct. 11, 2012.
Prior Publication US 2023/0054101 A1, Feb. 23, 2023
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 38/00 (2006.01); A61K 9/00 (2006.01); A61K 38/17 (2006.01); A61K 48/00 (2006.01); C07K 14/00 (2006.01); C07K 14/47 (2006.01); C12N 15/00 (2006.01); C12N 15/09 (2006.01); C12N 15/10 (2006.01); C12N 15/11 (2006.01); C12N 15/13 (2006.01); C12Q 1/02 (2006.01); G01N 33/50 (2006.01); A01K 67/0275 (2024.01); A61K 31/7088 (2006.01); A61K 31/7105 (2006.01); A61K 31/711 (2006.01); A61K 31/7115 (2006.01); A61K 31/7125 (2006.01); A61K 35/12 (2015.01); A61P 3/00 (2006.01); C12N 15/85 (2006.01); C12Q 1/6883 (2018.01)
CPC C07K 14/47 (2013.01) [A61K 9/0019 (2013.01); A61K 38/17 (2013.01); C12Q 1/025 (2013.01); G01N 33/5058 (2013.01); A01K 67/0275 (2013.01); A01K 2207/05 (2013.01); A01K 2207/10 (2013.01); A01K 2207/20 (2013.01); A01K 2217/206 (2013.01); A01K 2267/03 (2013.01); A01K 2267/0306 (2013.01); A01K 2267/0318 (2013.01); A01K 2267/0393 (2013.01); A61K 31/7088 (2013.01); A61K 31/7105 (2013.01); A61K 31/711 (2013.01); A61K 31/7115 (2013.01); A61K 31/7125 (2013.01); A61K 35/12 (2013.01); A61K 38/00 (2013.01); A61K 38/1709 (2013.01); A61K 48/00 (2013.01); A61K 48/005 (2013.01); A61K 48/0066 (2013.01); A61K 2300/00 (2013.01); A61P 3/00 (2018.01); C12N 15/85 (2013.01); C12Q 1/6883 (2013.01); C12Q 2600/106 (2013.01); G01N 33/5023 (2013.01); G01N 2333/47 (2013.01); G01N 2500/10 (2013.01); G01N 2800/56 (2013.01)] 16 Claims
 
1. A method of reducing TAR-DNA-binding protein 43 (TDP-43)-mediated neuronal cytotoxicity in a human subject suffering from amyotrophic lateral sclerosis (ALS), wherein the human subject does not have a mutation in a SOD1 gene, the method comprising: administering to the human subject a composition comprising a UPF2 polypeptide or a nucleic acid encoding the UPF2 polypeptide or a vector comprising the nucleic acid encoding the UPF2 polypeptide, thereby reducing the TDP-43-mediated neuronal cytotoxicity in the human subject, wherein the UPF2 polypeptide comprises the amino acid sequence of SEQ ID NO:4.