Gene therapy vectors for treatment of Danon Disease

The present disclosure provides gene therapy vectors comprising a polynucleotide sequence encoding a LAMP-2 polypeptide, methods of use thereof, pharmaceutical compositions, and more. In particular, the disclosure provides recombinant AAV vectors having AAVrh74 serotype expressing LAMP-2A, LAMP-2B,...

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Bibliographische Detailangaben
Hauptverfasser:	KERAVALA, Annahita, PRATUMSUWAN, Piratip, SHAH, Gaurav, WONG, Roderick, PRABHAKAR, Raj, YALAMANCHI, Naveen
Format:	Patent
Sprache:	eng
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING ORGANIC CHEMISTRY PEPTIDES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	The present disclosure provides gene therapy vectors comprising a polynucleotide sequence encoding a LAMP-2 polypeptide, methods of use thereof, pharmaceutical compositions, and more. In particular, the disclosure provides recombinant AAV vectors having AAVrh74 serotype expressing LAMP-2A, LAMP-2B, or LAMP-2C for use as a therapeutic in, for example, Danon Disease.