Self-regulating AAV vectors for safe expression of MeCP2 in rett syndrome

US11680275B2 · kind B2 · utility

Assignee

• University of Massachusetts · US

Inventors

• Miguel Sena Esteves · Westford, US
• Guangping Gao · Rosemont, US
• Michael R. Green · Geneva, US
• Dan Wang · Beijing, CN
• Tessa Mercedes Simone · Worcester, US

Key dates

• Filing date: Jun 6, 2018
• Grant date: Jun 20, 2023
• Expiry date: Aug 9, 2040

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2840/10
• WIPO field: Biotechnology
• WIPO sector: Chemistry

Abstract

In some aspects, the disclosure relates to compositions and methods of engineering a transgene. In some embodiments, the disclosure provides self-regulating recombinant nucleic acids, viral vectors and pharmaceutical compositions comprising a MeCP2 transgene. In some embodiments, compositions and methods described by the disclosure are useful for treating diseases and disorders associated with a loss of function mutation, for example Rett syndrome.