Abstract: This invention relates to polynucleotides encoding mini-dystrophin proteins, viral vectors comprising the same, and methods of using the same for delivery of mini-dystrophin to a cell or a subject.
Type:
Grant
Filed:
January 5, 2023
Date of Patent:
March 25, 2025
Assignees:
The University of North Carolina at Chapel Hill, Bamboo Therapeutics, Inc.
Inventors:
Xiao Xiao, Juan Li, Chunping Qiao, Scott W. J. McPhee, Richard J. Samulski, Maritza McIntyre
Abstract: This invention relates to polynucleotides encoding mini-dystrophin proteins, viral vectors comprising the same, and methods of using the same for delivery of mini-dystrophin to a cell or a subject.
Type:
Grant
Filed:
February 11, 2020
Date of Patent:
January 10, 2023
Assignees:
The University of North Carolina at Chapel Hill, Bamboo Therapeutics, Inc.
Inventors:
Xiao Xiao, Juan Li, Chunping Qiao, Scott W. J. McPhee, Richard J. Samulski, Maritza McIntyre
Abstract: The present invention relates to a modified FXN gene providing for increased expression of the encoded protein frataxin that can be used for treatment of Friedreich ataxia.