The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Jul. 15, 2025
Filed:
Dec. 20, 2019
The University of North Carolina AT Chapel Hill, Chapel Hill, NC (US);
The Trustees of the University of Pennsylvania, Philadelphia, PA (US);
Steven James Gray, Southlake, TX (US);
Erik Lykken, Dallas, TX (US);
Charles H. Vite, Philadelphia, PA (US);
Allison Bradbury, Philadelphia, PA (US);
The University of North Carolina at Chapel Hill, Chapel Hill, NC (US);
The Trustees of the University of Pennsylvania, Philadelphia, PA (US);
Abstract
This invention relates to polynucleotides comprising optimized GALC open reading frame (ORF) sequences, vectors comprising the same, and methods of using the same for delivery of the ORF to a cell or a subject and to treat disorders associated with aberrant expression of a GALC gene or aberrant activity of a GALC gene product in the subject, such as Krabbe disease (i.e., globoid cell leukodystrophy (GLD)).