Optimized slc52a2 polynucleotide constructs and expression cassettes
AssigneeBoard of Regents, The University of Texas System
InventorsSteven J. Gray, Xin Chen
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Abstract
Provided herein are polynucleotide constructs comprising codon-optimized SLC52A2 gene, vectors (viral or non-viral vectors) comprising the polynucleotide constructs, methods of using the polynucleotide constructs for delivery of an SLC52A2 polynucleotide construct to a cell or a subject, and methods of using the polynucleotide constructs for treating SLC52A2 deficiency such as Riboflavin Transporter Deficiency (RTD).
Claims24
A polynucleotide construct comprising a nucleic acid sequence comprising an engineered SLC52A2 gene sequence, or a cDNA thereof, or a variant thereof.
A viral vector comprising a polynucleotide construct comprising a nucleic acid sequence comprising an engineered SLC52A2 gene sequence.
We claim all methods, compositions, products and kits as shown and disclosed herein.
Dependencies are inferred from the claim text.