Engineered alpha-galactosidase a (a-gal a) peptides and functional variants thereof and associated methods of treating fabry disease
AssigneeSichuan Real&Best Biotech Co., Ltd.
InventorsBiao DONG, Siwu HE, Hoi Yee CHOW
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Abstract
Described herein are novel engineered peptides, viral vectors, and pharmaceutical compositions thereof, and uses of the same for increasing α-GAL A expression and for the treatment of conditions associated with α-GAL A deficiency, such as Fabry disease.
Claims32
An engineered peptide comprising or consisting of a portion of human alphα-galactosidase A (α-GAL A) or a functional variant thereof, and a portion of signal peptide of tissue plasminogen activator (sp21, sp18, sp20 or sp22) or α-GAL A (wt sp)-, or a functional variant thereof.
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A viral vector comprising an expression cassette comprising or consisting of a first nucleotide sequence encoding the portion of α-GAL A or the functional variant thereof, a second nucleotide sequence encoding the portion of sp21 or wt sp, or the functional variant thereof, and optionally a third nucleotide sequence encoding the portion of the cell-penetrating peptide or the functional variant thereof, and a promoter.
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Dependencies are inferred from the claim text.