US 12,202,867 B2
Adeno-associated viral chimeric TDP-43 proteins
Philip C. Wong, Baltimore, MD (US); Aneesh Donde, Baltimore, MD (US); Jonathan P. Ling, Baltimore, MD (US); Liam Chen, Baltimore, MD (US); and Mingkuan Sun, Baltimore, MD (US)
Assigned to THE JOHNS HOPKINS UNIVERSITY, Baltimore, MD (US)
Appl. No. 17/287,730
Filed by THE JOHNS HOPKINS UNIVERSITY, Baltimore, MD (US)
PCT Filed Nov. 4, 2019, PCT No. PCT/US2019/059623
§ 371(c)(1), (2) Date Apr. 22, 2021,
PCT Pub. No. WO2020/093035, PCT Pub. Date May 7, 2020.
Claims priority of provisional application 62/754,791, filed on Nov. 2, 2018.
Prior Publication US 2022/0275034 A1, Sep. 1, 2022
Int. Cl. C07K 14/47 (2006.01); A61K 38/00 (2006.01); C12N 15/86 (2006.01); C12Q 1/6883 (2018.01)
CPC C07K 14/4702 (2013.01) [C07K 14/47 (2013.01); C12N 15/86 (2013.01); C12Q 1/6883 (2013.01); A61K 38/00 (2013.01); C12N 2710/10343 (2013.01); C12N 2840/44 (2013.01); C12Q 2600/118 (2013.01); C12Q 2600/156 (2013.01)] 10 Claims
 
1. An adeno-associated virus or vector that further comprises a non-native nucleotide sequence capable of expressing a chimeric protein comprising an N-terminal nucleotide binding domain of transactivation response element DNA-binding protein (TDP-43), a C-terminal domain derived from a splicing repressor, and an autoregulatory element, wherein the autoregulatory element comprises SEQ ID NO: 3.