Adeno-associated viral chimeric TDP-43 proteins

US12202867B2 · kind B2 · utility

Assignee

• THE JOHNS HOPKINS UNIVERSITY · US

Inventors

• Philip C. Wong · Timonium, US
• Aneesh DONDE · Baltimore, US
• Jonathan Ling · New Brunswick, US
• Liam CHEN · Baltimore, US
• Mingkuan SUN · Baltimore, US

Key dates

• Filing date: Nov 4, 2019
• Grant date: Jan 21, 2025
• Expiry date: Jan 25, 2042

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12Q2600/156
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

An adenovirus or adenoviral vector is described that includes 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. Methods of using the adenovirus or adenoviral vector to treat degenerative diseases such as inclusion body myocytosis, amyotrophic lateral sclerosis, and frontotemporal dementia are also described.