Compositions and methods for gene therapies

Abstract

Provided herein are polynucleotides comprising a nucleotide sequence encoding a biologic molecule (such as human α-galactosidase A), a nucleotide sequence encoding a promoter (such as a hepatocyte-specific promoter or a hepatocyte-myocyte bispecific promoter), or a nucleotide sequence encoding an expression cassette (such as a human α-galactosidase A expression cassette). Also provided herein are promoters, expression cassettes, vectors, host cells, gene delivery systems (e.g., recombinant viral particles such as recombinant adeno-associated virus (AAV) particles and non-viral gene delivery systems), associated pharmaceutical compositions, as well as methods using the same. Such compositions and methods are particularly useful for gene therapies, especially for lysosomal storage disorders including Fabry Disease.

Dependencies are inferred from the claim text.