Optimized GALC genes and expression cassettes and their use
AssigneesThe University of North Carolina at Chapel Hill, The Trustees of the University of Pennsylvania
InventorsSteven James Gray, Erik Lykken, Charles H. Vite, Allison Bradbury
Life of this patent
Estimating the term…
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)).
Claims26
A polynucleotide comprising a canine or human GALC open reading frame, wherein the GALC open reading frame is codon-optimized for expression in a canine or human cell, wherein said canine or human GALC open reading frame comprises the nucleotide sequence of SEQ ID NO:1 or SEQ ID NO:2, or a nucleotide sequence having at least about 90% identity thereto.
Dependencies are inferred from the claim text.